would this pill help peoplewith tourettes syndrome?
Please keep researching there are many people with this very distressing condition at present there is no cure.

Rose, London, England

I think this is wonderful!! I do wonder if there is hope for cures for missence mutations like MCADD. This is what my child has.

carrie, greenville,

It is the most encouraging medical news. Please let me know whether this drug will be the answer for a cure for Huntingtosn Disease.

Anne, Colombo, Sri Lanka

How will this drug hllp a person with mjd out. their mutated gene produces a toxic protien to the brain and i do not want the process sped up bu repairing the long strands of the toxic protien.

Dimas Lindo, Swansea, USA MA

With mjd can this drug find the difference between a good protien producing gene and a toxic more dominate toxic protien producing gene ? If so how can it? I would like to refer this info to my Doctor for reference.

Dimas Lindo, Swansea, USA MA

My 2 brother in laws are affected by Becker Muscular Dystrophy they struggle to earn a living for themselves and their families in spite of their health problems. They work hard and pay their taxes like everyone else - oh sorry that does not include the growing minority of FULLY able bodied but down right lazy, scrounging 'do anything but a days work' sufferers that are quite happy to sit on their backsides and take benefit payments for fake incapacity and invalidity. Its a shame that no one has discovered that being morally bankrupt is a product of 'dodgy DNA' and we could add that to the great clean up of the human race - I know what about creating a pure master race - heard that before somewhere ( ? ).
Surely advancements in medical science that improve, heal and saves lives can only be welcomed.

SJ, Yorkshire, UK

Could this work with Wiscot Aldrich protein mutation, causing very low neutrophils count?

Akis, Limassol, Cyprus

I am suffering from XP 21 Myopathy, wil it help in that also.

And will we able to get this only in 2009 not earlier that

sudha, delhi, india

Would this help with FSH muscular dystrophy?

Lisa Saunders, Vidalia, LA 71373 USA

My daughter has NF2, and the cause is a spontaneous genetic mutation on C 15. She does have a nonsense mutation, which results in a stop codon. Currently she has, of course, bilateral acoustic neuromas and innumerable CNS tumors of brain and spine, along with small subcutaneous schwannomas or neurofibromas. I would like to know how to get her involved in this research ASAP, please!!! She is 20.

Thank you!

Lianna Nix, Monticello, USA/ GA

Hi Joe, from Chapel Hill: Your astute comment about CF and dysentery falls through pretty quickly with just a little additional thought. Your implication is that being protected from dysentery by having the CF gene confers a survival benefit (at least 'till you can reach reproductive age). If that were true, the CF gene would be passed along more frequently in populations at greatest risk for dysentery. In fact, the opposite is true. The CF gene far more prevalent (3X) in people of northern european heritage compared with african. Apparently diversity isn't doing us any favors after all in this regard.
Jenn, Norwich VT

jennifer keller, norwich, vt

Can PTC124 be used to treat Chronic Immune Dysneuronal Polyneuropathy (CIDP).

D K Holub, Torrance, CA

Shirley, not all genetic disorders are caused by a 'nonsense' mutation. 'Nonsense' mutations cause a premature stop, whereas a 'missense' mutation can cause an amino acid change resulting into a genetic disease like thalassaemia. The whole gene itself can be deleted, or there can be an insertion or deletion into the gene. Also, it can affect a 'regulatory element' of a gene, for example in genetic forms of cancer the 'normal' protooncogenes that control cell growth become mutated into 'oncogenes' that lets a cell grow abnormally resulting into a tumour.

Sama, Edinburgh,

I have an adopted daughter with a genetic disorder called MCAD. MCAD is passed in an autosomal recessive manner, like a great number of genetic disorders. That means both of my daughters birth parents carried one mutated gene. They weren't affected by the disorder and had no idea they were carriers. There was a 25% chance my daughter would inherit both her parents healthy genes and not have the disorder. There was a 50% chance she would inherit only one mutated gene and be a carrier. Unfortunately, she also had a 25% of being affect by the disorder.
If you want to clean the gene pool, you'll have to sterilize all the carriers of disorders as well as those affected. Statistics say that 1 in 50 people have the MCAD mutation. That is alot of people to sterilize, and that would only rid us of one disorder. Do we really want to go there?

Becky, Madison, AL

What percentage of cystic fibrosis patients have a "nonsense" gene? We always understood that all cf, and all genetic disorders, were caused by a "nonsense" gene, but the way the article is worded, it sounds like only five to fifteen percent of each of the genetic disorders like cf and md are caused by a "nonsense" gene.

Shirley, Elgin, TX

I have 2 grandsons with hemophilia and this article was an answer to many, many prayers. WHere can I buy stock in this company???
Carol Lizana, Baton Rouge, LA.

Carol J. Lizana, Baton Rouge, LA.

Well, I am astounded by the ignorance and lack of compassion of the dissenters in this correspondence. Many of the conditions targeted by PCT 124 are caused by recessive genes carried by about 1 in 50 of the population in the UK.

eg Oxford has a population of approx 617,000, 12,340 people will carry the defective gene for a single genetically determined condition.

When both parents carry the same recessive gene there is a 1 in 4 chance that a child will develop the condition, or be a carrier, 1 in 2 will not being carriers .The chance is the same in each pregnancy.

It is only when a child is born with the condition that the parents know they are carriers and one of the grandparents from each parent and maybe other siblings. Theirs and extended families future hopes are dashed and their lives never the same again. They are innocent victims of a wildcard genetic match.
It may be you tomorrow WAKE UP , join the real world learn some compassion or is that too genetic?

Sue , Bordon, Hampshire

I have a daughter with CF and I read this article with increasing amazement, it is what I have hoped for all her life, that a break through would come before it was too late to benefit her. Yesterday she had a routine clinic appointment with a specialist CF team, at the end of the appointment I asked about this treatment and was laughed at, they asked if it was April 1st or perhaps something from a local paper, not one of the three doctors present had any knowledge of this. Is the reporter being over optomistic or should I move house to another heath authority?

Jampanda, Downham Market, Norfolk

Wounderful news!, however I cannot believe some of the comments posted here.
A geneticist once told me everyone has at least 6 genetic abnormalities, if you are bald or are colour blind it is due to a genetic condition.
As for the cost of keeping sufferers alive we are quite happy to spend money on a Trident replacement, a rubbish NHS computer system (which I understand costs equivilent to the entire NHS budget), and ID card systems/traffic charging schemes. I would certainly prefer to keep around the children and adults who suffer from genetic illness than any of these pathetic white elephants.
If this drug only saves only 5% of children with Duchenne or Cystic fibrosis it will be a remarkable breakthrough, this is a first step, more developments will follow.
This is a small ray of hope for families who have been living in a veil of darkness, I wish every success to the team at PTC, and others working to eradicate the effects of these terrible conditions.

Bob Foster, Sittingbourne, Kent

Sounds fantastic, though a little more care with the journalism would have been nice. This drug only treats one form of mutation - addition. It doesn't do anything for substitution and deletion - much more common forms of genetic mutation. Certainly it looks as if it has the potential to reduce the pain in many people's lives.

Barry, the reason why these diseaes have only appeared in the last hundred years or so is becuase a hundred years ago a CF suffer would have been unlikely to live more than a few years, the same applies with a lot of the other diseases. Thanks to the developments in medical science that have already taken place, these people can now live for an increased amount of time, so people actually take notice.

Kris Jones, Cambridge,

Arguments like that of the original poster, Eric, Laurel, MD are based on the fallacy that the human race as a whole is better off if each individual is in optimally fit. Setting aside any moral issues with pursuing that policy, consider that a more diverse population is more likely to contain an individual with a chance mutation that confers immunity to some new disease. The two examples I can think of are cystic fibrosis - dysentery and sickle cell disease - malaria. In each case, having the first disease (or at least having the mutation on one chromosome) confers some immunity to the second disease.

There is another, more subtle, reason why the argument is incorrect. Suppose, for example, that there is a combination of 3 mutations that confer an advantage (better memory, stronger muscles, whatever) to people who have all three, but that any one of those mutations would prove fatal. If we can survive the deleterious mutations, then we have a chance to find the good combination

Joe, Chapel Hill,

Your telling people born with a genetic disorder to not be allowed to have children? yeah that makes sense. How about we round up poor people and do something with them for being poor right?

Joe, columbus, Ohio

would this pill help children with rhett syndrome.?

geoff, birmingham, uk

It's a science issue, not a heart issue. The mutated gene that causes my daughters disorder was passed to her by her birth parents. It's called an autosomal recessive pattern.They each carried one gene mutation and when my daughter was concieved, she received both mutated genes. There was an equal chance of her receiving theit unmutated genes and not having her genetic disorder. There was a better chance of her only receiving one mutated gene, and being a carrier herself.
The point is that to wipe out a vast number of these disorders, you have to keep carriers from reproducing with other carriers. Statistics say that one in fifty people are carriers of my daughter's disorder. All of those people would have to be sterilized. That is A LOT of people, and that is only one disorder.
We'd all have to be tested for every genetic mutation and by the time we were done, I doubt anybody would be fit to reproduce. And I don't think we want to go there!

Becky, Madison, AL

It is sad to see a modern call for eugenics by withholding a potentially-life-saving drug. It is disturbing to see someone referring to themselves as a physician state that "people with serious genetic disorders should not propagate". It is most upsetting when the statement is filled with ignorance.
Most of us (including the individual stating “people with serious genetic disorders should not propagate") carry on average about a half dozen genes for lethal genetic disorders. For most genes, we carry two copies (one inherited from each of our parents) and a lethal mutation in one copy does us no harm. The writer wishing to end the reproductive plans of those with “serious genetic disorders” is punishing those that merely have the misfortune of having two defective copies of the SAME gene, not some kind of genetic inferiority. Some day perhaps we will be able to measure the number of defective genes a person does carry. We would then have perfect data upon which to base a eugeni

Robert J Vosatka, MD, PhD, Kinnelon, New Jersey

Leslie makes unsupported sweeping statements that indicate her medical knowledge is deficient. For example, fragile X is more complex than she indicates and I've found no research that indicates it's transmission is age dependent. Rather, a fragile X male will transmit a faulty X chromosome to all daughters but cannot transmit it to sons. Females with one copy of the fragile X chromosome can transmit it to both sons and daughters. In this case the son or daughter has a 50% chance of inheriting the fragile X. Perons with fragile X should seek genetic counseling.
And why can I speak about this subject? Because my sone has fragile X and I've been learning about it for almost 20 years.

Richard, Portsmouth,

I have a daughter (adopted) with a genetic disorder (MCAD). This pill could possibly help her. At least it offers hope.
She's nine months old, and hasn't really lived an unatural lifestyle. A genetic mutation caused her disorder at conception. That was long before she had the chance to doing any unhealthy living!
As far as manufactureres lining their pockets... my daughter is already taking medicines daily. I'd gladly switch from a medicine that helps her symptoms to one that erases them! We'd probably save money by eliminating hospital visits!
And while we're at it... let's go ahead and sterilize her. Of course her birth parents are carriers, they could produce more children who will weaken the entire race, so they'll need to be sterilized too. There are estimates that 1 in 50 people are carriers of the MCAD mutation so we'll have to test everybody and get busy with that sterilization program. I'm not sure your idea would leave anybody able to reproduce!

Becky, Madison, AL

If this drug does what you say it does, it should be rushed to Stephan Hawkins before it is too late.

Herbert Kaufman, Plainfield, New Jersey/USA

Holy moley. "Dodgy DNA weakening the entire race"?! Forced sterilization? I thought we'd debunked eugenics (genetic purists) sixty-two years ago. Eric from Maryland and Jamie from England, you guys have a sick understanding of evolution.

Different genes are not "bad" for human evolution. There is no ideal human being. The truth is, a diversity of genes will help us as our environment changes. We are not some breed of flower to be bred for a single trait (and subsequently wiped out by a single disease).

This medical advance is not a backwards step in a supposed "ladder of evolution". Humans have evolved relying on technology and civilization to survive. Do you think we would last long if we stopped using our brains and compassion to help each other live and thrive? This is a step forward. For all of us.

Ben Wong, Seattle, WA, USA

I think this breakthrough is fantastic. However wonderful the life saving effects this drug brings, the people who carry the gene that is responsible for these mutations should seriously consider the reprocussions of passing those genes to another generation.

Peter, Fort Myers, FL

I am a sufferer of the condition CMT Type II (muscular atrophy). It has been a condition that has run in my family through generations and has mainly occurred in the males. Although in recent years, it appears to have now changed tack to affecting some females in the family (including myself). I am excited by the news regarding PTC124 and hope that this new drug will be answer to people's prayers.

Wendy, Birmingham, UK

its good but for which stages of the diseases does it work for, there must be some side effects what are they? does it work for everyone for example every race has different genes does it work for all of them, who has it been tested on? would it be available worldwide for example will it be available in LEDCs? what if the person who needs to use this drug also has another disease for example epilepsy? will it work with the drug used for epilepsy? will it worsen epilepsy, will it cure epilepsy? if it works for everyone then all our prayers will be answered

Jamila, Leicester,

what about hermansky pudlak syndrome?? also toutette syndrme??

naio, bronx, newyork usa

This reminds me of the era of Penicillin serendipity, during the world war 2, I felt the same happiness when my pharmacology Professor was teaching us about discovery of Penicillin and he said for the first time in human history " Life was used against life to save life". I was delighted to hear those words and could understand the gist of what he was trying to tell us. While reading this article I again experienced the same ecstatic feeling. A truely wonderful experience.

Mohit, Gurgaon, India

Paul from Cambridge (or anyone else), can you say whether or not this drug would be useful against Myotonic Muscular Dystrophy Type 1? Is the CTG repeat of MMD1 a "nonsense" mutation? Thanks for any help.

Hua Kul, Cleveland, Ohio, USA

is there some hope for ppl with tourette syndrome amd with hermansky pudlak syndrome???

noai, newyork, usa

1) As long as they don't correct genetic disorders like standing up for what you believe in etc and the colour of your eyes it sounds interesting...
2) How much will it cost for the course of the patient's life? Will this evolve into a pill that corrects the erronus code permanently or will it remain a pill to fix on an expensive temporary measure so that you are trapped paying for it (assuming you can afford it/or not) for the rest of your life?

Jason Smith, CANTERBURY, UK

Jackie you tell Richard that "embryonic stem cell research IS legal. The debate is only whether or not federal funding is provided. " Yet another uniformed Texan. The President has made it illegal for the Gov to invest in stem cell research, private companies and individual States with their own budget can do whatever they like, as long as it ethical. I work with several companies doing very nice work in stem cells... please stop believing your pastor and your president, they are both uniformed alarmists who think they can answer any question by quoting scripture...they are, like you, plain wrong!

Beemer, Boston, USA

"PTC124, which is made by PTC Therapeutics, has been staggeringly successful in animal models."
Of course people who are against experiments on animals should not be allowed to take this medicine.

Hans, Noordwijkerhout, Netherlands

After speaking to my CF consultant yesterday, this news should be treated with great caution. In the case of CF, for example, the claims made in this article are enormously exaggerated: only a very small percentage of CF cases are caused by a nonsense mutation, and where research has been carried out with similar drugs they have had little conclusive effect. I don't know the circumstances for other conditions mentioned here.

Tim, London,

But does it work for Pseudohypoparathyroidism Type 2?

Chad Singer, Cleveland, Georgia

Its unfortunate that people have these diseases and i'm sure that your sons, daughters, husbands lead very fulfilling lifes. So are you saying with 100% certainly that they would not pass on their faulty genes in 100% of cases? If that is the case and is proven then i'm sorry.

However how many of these unfortunate use large amounts of expensive medical treatments? And I'm sure i'm right in saying that they will rarely generate enough tax to pay for them and required care. Which is fine how things are, but the more is spreads eventually this wont be the case. Just use your brains and not your hearts of once!

Jamie Brown, Maidstone, England

Responding to Jamie: As for your disbelief that a previous writer could endorse enforced sterilization, because "we don't live in a police state." Two things - one is that you're misrepresenting what he said. The other is that he's writing from the USA, as I am, so he does live in a state where total surveillance and punitive social controls are more and more a reality rather than imagination. Now that I think of it, haven't I heard that Britain has moved in much the same direction? Regardless, Eric in MD is championing a world where brutal, debilitating diseases are allowed to run their course and ruin lives that could be happy ones. I imagine he's affluent and healthy, one of the Americans who, unlike myself, have health insurance. In my experience, that's the social profile of most people who have this kind of brutal, supposedly Darwinist ideology. Pure bloodlines rightfully rule, yes? How dare you, says he! Health care is not a class issue!
Best to you both.

Eric, Denver, CO

Is the bipolar condition, as experienced by Stephen Fry and many others likely to be helped by this treatment with PC124?

Kevin Whittal, Ledbury, UK

Marvellous news for the afflicted individuals and their families and friends. Thanks to the scientists for a intellectual and technical tour DE force. Another milestone for human's endeavour to conquer all diseases. But can we see a double edge sword and a time bomb emerging in the mist of our euphoria and jubilation? What are the consequences and ramifications if we can push the frontier of medical advances to the point where all incurable diseases are irradiated and we are in a position to prolong lives indefinitely? In order not to become the ultimate victims of our own success we are face with several enormously difficult choices. Namely (1) stop all medical research work (2) make sure our space exploration programmes are in step or ahead of medical advances(3) compulsory limitation of population growth(4)
voluntary or involuntary euthanasia(5)sequential death and resuscitation of individuals. I am glad I won't be around to see the horrible steps mentioned above implemented.

Wing, Poole, UK

"If a faulty gene gets passed on more frequently, then in a hundred years, it could easily go from a tiny percentage of children being born with the disease to a majority."

This logic is flawed in two ways.

1) Certainly we would not expect the "flawed" version of the gene to rapidly spread to a "majority" of the population; there is no selective pressure to cause this to occur.

2) This comment presumes that the state of medical technology will remain static for the next century. While we may not be able to CURE these diseases in the next several years, I think it is rather pessimistic to assume that we will NEVER be able to do so.

Tom, Seattle, WA

WOW, there are some crazy comments here, and so cruel to those of us suffering. Blaming our diets, our beliefs, and challenging our rights to parenthood. Everyone has a RIGHT to say what they like (as I am) but why be cruel, demoralizing, or harsh? its not necessary and actually makes YOU the commenter look bad and ignorant. I am EXCITED to see research moving forward. Yes 5-10% is a small number (if you wonder if it might work for you you need to have a DNA mutation analysis done and talk to your doctor) but to those 10% its means the world! And it gives the rest of us hope for more research breakthroughs. Stem cell research is important but did you know that we adults carry stem cells in our bodies that could be used? and that fetal cord blood (from the umbilical cord at birth that is just thrown away) also holds stem cells? there are other sources for it then killing embryos. May God bless all those who are suffering and those searching to help!

Jennifer, Hillsboro, OR

Reply to Eric Laurel:

Your claim that a faulty gene could in a hundred years go from rarity to majority is a gross misstatement. Take for example a disease allele that has an incidence of 1% in the population. For natural selection to reduce this allele frequency from 1% to 0.1% would take 900 generations. Furthermore, even if this selection process were relieved by a drug, that doesn't mean the disease allele will then be positively selected for: by Hardy-Weinberg principles it would remain at its present frequency.

Kate Sevigny, Fremont,

Some of these commentors are not thinking clearly. The only thing that influences the persistence of a trait in a population is its effect on reproductive fitness. Any disease that has onset following reproduction is not in the least affected by genetic selection [look at baldness]. We are in no danger of breeding a population that is dependent on one of these drugs.

Michael P, Irvine,

Eric, do you have any idea how many generations are required to effect any sort of real evolutionary change in a population? There is no way for genetic disorders that are currently very rare to increase in prevalence such that it will represent a majority of new births in just 100 years. Although these individuals will no longer be NECESSARILY excluded from the genetic pool, it is still not the case that these disorders will be selected for in an evolutionary sense, and even if they were, it would take FAR longer than 100 years in order for there to be any discernable impact on the general population.

Furthermore, people with these diseases are responsible individuals, who would take the likelihood of transmission into account prior to the decision to have children. I can only assume that they would not choose to have children if it was likely that the child would suffer from the disease and/or that current treatments wouldn't be able to efficiently and effectively treat.

Jenny, Chicago, IL

will this new drug help the genetic disease called huntigtons chroe? i ask this question as i have lost my mother to this illness and my brother recently has developed the disease, we are looking for something to help him as he is only young.

janet taylor, warrington, united kingdom

Of course, that is a pretty narrow view (Jamie Brown's), it assumes that we wouldn't ever have the ability to ultimately repair our DNA to weed-out nonsense mutations down the road. I'm betting at some point we will.

Chandler Hale, Tulsa, OK/USA

Is spinocerebellar ataxia #6 or any of the SCA's included in this group?

Pat, Houston, TX

This took my breath away. Amazing.

thepseudoshrink, Manila, Philippines

I told my eight-year-old granddaughter who has CF about this drug and the coming clinical trial. She cried for joy and wanted to know how to join the clinical trial. She said, "Tell them I'll be there." So, is it too late to take part in the clinical trial, and, if not, what are the qualifications to do so?

Shirley, Elgin, TX

Does anyone know if huntingtons disease benefit from this drug please

Debra, Longford, Ireland

Genetic disorders such as Duchennes MD, hemophilia, Alzheimer's, autism, schizophrenia, cancers, diabetes type 1 , MS, all non-familial version could be reduced in the first place if men knew to father all their babies by the age of 33. They could also cryobank sperm in their mid 20s for later fathering of babies. The pills sounds great, but it would be best to prevent paternal age derived genetic disorders.

Leslie, El Ceritto, ,

I'll bet it won't amount to anything, most likely a pump and dump scheme for the biotech stock of the underlying company. There was a compound called CPX some years ago that an American company, SciClone Pharmaceutical, touted as a CF cure. Nothing came of that as it was found that what worked in an animal model did nothing in humans. Plus, as someone with the delta-F508 cf mutation, this does nothing to solve that problem, which is the genetic mutation that is the cause of 70-75% of CF. Perhaps it will work for MD, but even for those folks, I'd warn them to not get overly excited until studies come out.

John Smith, jacksonville, USA / Florida

Even if this pill is not a cure, it can certainly sustain a good quality of life until the cure comes. The hope for the future of humanity is not sterilization of people with genetic problems, it is finding a cure for those problems - finding a way to normalize the problem gene. Until then, lets hope the pill works as well as its most optomistic fans believe it will.

Eugene Denson, Alderpoint, CA

It would also be great if all men knew about the male biological clock and how mutations in sperm making cells increase by the age of 33-35 causing autism, schizophrenia, diabetes type 1, MS, cancer, Alzheimer's, and then in the son's of daughter's of older fathers Duchenne's, hemophilia, fragile X and probably autism all de novo/ non-familial.

Leslie, El Ceritto, ,

One step at a time. Noone can decide who has more right to health or life. We work around the problem now, and we can still fix it later. Considering which humans have the right to breed is a dangerous and ugly road to travel down.

Nathan Kinsman, Portland, Oregon

I always wanted to take a course like this when I was young and find cure like these, too bad as much as I try I am limited to self study. Thank God people like you have the dream to develop such, may it be for your selfishness or the the desire to help others it is good news people like you exist in this lifetime. BRAVO!

Adrian, cebu city, philippines

Mr Jamie Brown - How dare you say that sufferers of the above mentioned diseases be sterilised for the good of the human race?

I can only presume you lean towards Nazi tendencies.

Why should my daughter be stopped from having children if she so wishes? There are tests available now that can tell if the baby will be born with CF, something that wasn't available many years ago when we had her. How about checking the facts out for the chances of having a child born with CF before you jump to any conclusions.

I am guessing you also want 'Britain for the British' too

Fenton R, Manchester, UK

Does anybody know how this drug allows the ribosome to skip unwanted stop codons but still respond to the real stop codon? It seems like you would get alot of proteins that are too long with this drug.

Chet, Salina, KS

As a truly lay person, I've heard of breakthroughs in the past. Should I be optimistic here?

Sian, Bradford, UK

my 42-yr old husband has Fascioscapular-humeral muscular dystrophy.The hope that this drug could potentially bring to him,and to thousands like him,is astounding! My hope is that this drug will be available soon enough to make a differenc in his and in other's lives SOON.Watching this good man being consumed by MD,little by little,is its own unique form of Hell.Looking forward to seeing more on this--

janell , Babbitt, MN

My wife forwarded this information to me, and we are amazed. My daughter has Rett Syndrome. Thanks to the work of Dr. Huda Zoghbi at Baylor Medical School in Houston the gene causing Rett Syndrome has been identified. Our daughter has a nonsense gene that has caused Rett Sydnrome. We are so hopeful that this medical treatment proves promising. I hope we can try it soon. Thank you to all of you researchers who devote your lives to improving the lives of others.

Clifford Fry, College Station, Texas

I have to agree with Jamie Browns comment, in that fixing a genetic disease will only perpetuate it. If a faulty gene gets passed on more frequently, then in a hundred years, it could easily go from a tiny percentage of children being born with the disease to a majority.

Would we really want future generations to largely depend on a daily drug in order to survive? In a sense, medical science, while definitley good for the individual, can have negative effects on the human race in the long run.

People with serious genetic disorders should not propagate, and risk passing those genes on to future generations. To do so is dangerous and selfish.

Eric, Laurel, MD

Reply to Jamie Brown of Maidstone:

I cant believe that you are even entertaining the notion of enforced sterilisation, get a life. Last time I checked we dont actually live under some military state!

Harry, Leeds, UK

My brother has Cystic Fibrosis and will be celebrating his 40th birthday this June. When he was born, the life expectancy was not even into the teens, and now many patients with CF often live a fulfilling life through the miracle saving advances in medicine.

This is one awesome step towards a completely fulfilling life for sufferers of genetic diseases. Unfortunately the side effects of CF has disabled my brother even more with Diabetes, which led to blindness, liver failure and other side effects. But if this solution keeps the other effects at bay, it is a great day for science.

David Crowley, Atlanta, GA, USA

Jamie Brown, get yourself sterilised. You carry as much dodgy DNA as anyone. Do a course in basic genetics, start with spontaneous mutations. It's attitudes like yours that weaken a race. My 23 year old son, who has DMD, manages his illness and leads a very full life. He cotributes more to society than many able-bodied persons and he doesn't worry about dying just yet. He is too busy living. People live with all sorts of illnesses and it is great news when there is any new treatment. Look at Stephen Hawking and what he has done in his life.

Siobhan Jowett, Thoiry, France

This morning, having read this in the Times, I was delighted to think that my daughter's CF could be managed more effectively.
This afternoon, after investigating her gene mutation, it seems this will not help her. Along with 79% of other sufferers in the UK, she has df508 which is not a nonsense mutation (it is deletion or missense, not entirely sure).
It is fantastic news for those with other mutations, and many congratulations to the scientists, but I am disappointed that the reporting made out that it was a blanket solution for cystic fibrosis when in fact it will help a minority of patients.

Heather, Oxfordshire,

Is 'PTC Therapeutics' traded on the stock market ? Or private setup within the university......

A Hunneybell, Warwick, UK

I wonder if this drug could offer any hope to people with genetically inhereted skeletal dysplasia's...such as Jeunes Syndrome.

Mike, Wetumpka, Al

I think this is an amazing breakthrough. I am a Critical Care Nurse and see the suffering my patients with these disorders go through. I hope the people who have been offended will look past that and focus on the positive.

wanda keith, indianapolis, IN

From your mouth to God's ear ! Good luck and God Bless.

Dorothy, Staten Island,New York, United States

I too am appalled by Jamie Brown's comments. I am married to a sufferer of a muscular dystrophy. We have 3 wonderful children, born before my husband was even diagnosed and they in no way "weaken the human race" and do not have the condition or carry it. How dare you even suggest sterilisation. Even those with genetic disorders have the right to live a life without prejudice from the likes of you. This is a fabulous breakthrough for people who suffer and this day should be celebrated.

Denise, Cheshire,

This is very positive news but it has to be stressed it is still in a trial stage; hopes can be raised a little further at each successful step.

Good luck with the research.

As a married person with a related muscle condition I am personally outraged by Jamie Browns comments. Such misinformed and uneccessary thoughts contribute to a daily tide of discrimination faced by my peers and I. On a positive news day keep it to yourself please.

Dan, Cheshire,

Barry's comments below are extremely uninformed. As one of the conditions this drug helps mitigate, Muscular Dystrophy is entirely genetic in origin. It is just completely wrong to imply that my wife who has always exercised and has ascribed to a healthy diet all of her life is somehow responsible for the unfortunate condition of our son.

Also, without medicine variants such as PTC124, my son will most likely be dead in 20 years without having experienced the same quality of life as his friends, through absolutely no fault of his own. I can only hope he has the "stop codon" sequence that this treats - hurry up, DNA test!.

A pill a day to see my 4-year-old son be healthy versus struggling to breathe at 16 because his chest muscles are deteriorating? Good lord, sign me UP!

Chris , Canada,

Although Duchenne affects males, it is not passed on by males, so Jamie Brown should not worry. Jamie may not have relations with this problem so he may not understand the enormity of success in this field for effected families

Carmel, Victoria Gozo, Gozo Malta

Available in three years? On the NHS it will not. Due to the high cost of the drug, the committee responsible for drug
prescribing on NHS, will procrastinate for a year or two, then, under instructions from the Ministry,one will see yet another post code lottery.

Don Ralph, Alicante, Spain

Never mind ''curing'' genetic conditions, will it allow me to run, cycle or swim ,faster, further than anyone else without detection?

Tom Sedman, Worcs,

All of the below need to learn a little science. These diseases; cystic fibrosis and Duchenes MD are monogenetic diseases. They are NOT preventable and are untreatable. The people that have these diseases are probably the most wothwhile to treat because they have not lived a lifestly which has caused them to develop the disease. Furthermore they are not epigenetic.

Z Etheridge, Oxford,

Great success.....Good luck to the people with that disease..

JaeGoo Lee, Seoul, Rpulic of Korea

This leads one to think. Maybe a sufferer could lead a normal life with this, get married, have kids and pass on the dodgy DNA weakening the entire race. These sort of drugs should only be used if the sufferer is sterilised.

Jamie Brown, Maidstone, England

A couple of points on previouse comments.

Walt, PTC124 was developed to treat disorders that are due to nonsense mutations in genes, Duchenne muscular dystrophy and Cystic Fibrosis being the first two examined. MS is an autoimmune disorder so it's extremely unlikely that PTC124 would help.

Yuu, you're right that if this drug also caused the translational machinery to also read through normal stop codons in mRNA it would cause serious problems. PCT have examined whether this happens in vitro, in animal models and in early clinical trials in humans and concluded that it does not appear to affect stopping at normal stop sites. So it looks OK at the moment.

Of course all these early studies are relatively short term, and problems might arise when this drug is taken for many years. This drug is intended to treat life threatening and very dehabilitating conditions so I think that many DMD and CF sufferers will see the risks as worth taking.

Paul, Cambridge, UK

This is a fantastic piece of work by PTC Bio - who have screened thousands of chemicals to find one that skips stop instructions safely. Sadly only (10-15%) patients with genetic errors that create stop instructions mid-way through the gene can benefit from this drug. The majority of patients have deletions and so cannot benefit from PTC-124. However, it means that people with alot of genetic conditions will need to be tested to see if they can benefit from PTC-124.

In the UK boys with Duchenne Muscular Dystrophy are being registered for PTC-124 as well as exon skipping and other potential drugs such as Utrophin upregulation on the DMD Registry (http://www.dmdregistry.org).

Robin, London, England

James, as someone with CF myself, I'm already on a lot of 'crap' for the rest of my life. Trust me, cystics aren't generally that bothered about taking another daily pill on top of all the rest, as long as it works. We only seem to be able to find band aids at the moment, but if they keep getting better, I'll take 'em.

(Now if government could do something about granting us free prescriptions as they do for other chronic diseases...)

Tim, London,

I'm underwhelmed by this article. A pill (that will no doubt make billions for its manufacturer and be expensive for the taxpayer who funds the NHS) to 'cure' diseases that are already very easily prevented.

Practically all the chronic degenerative diseases we see in Western society today were unknown or extremely rare until the last century. These are not 'genetic', although they could be epigenetic (look it up). They are caused by our unnatural lifestyle.

And the most unnatural aspect of that lifestyle is our 'healthy' diet. At no time in our evolutionary past did we eat the carbohydrate-rich, unnatural-fat diet we do today. For practically the whole of our existence we ate a largely carnivorous diet. There are still many cultures we call 'primitive' who still do -- and don't get any of the diseases we suffer.

There is very little evidence that diseases caused by unnatural lifestyle are helped by drugs. But drugscertainly improve the health of drug companies' bank balances.

Barry Groves, Oxford, UK

I think this is brilliant news. I have severe rheumatoid arthritis, and if I found this gene had been passed on to my grandchildren, I wouldn't hesitate for a moment in recommending they take this 'pill'. As for taking this 'crap' for the rest of your life James, just consider what crap patients who have incurable diseases take - steroids, anti-inflammatories, anti-tnf. I know what I'd rather take if I had the choice.

Norah Fisher, Worthing, UK

Would this drug aid huntingdons disease?

RP, slough,

We along with the parents of the other 7000 children and young adults with Cystic Fibrosis in the UK have woken to a new dawn if this drug does what it says on the tin.

The race is now on to make it widely available before our children have degenerated too far in their conditions for it to be of benefit. I hope that the government will put all available resources into the necessary addtional trials that may be necessary and get it out there asap.

This is not just a matter of saving lives from a debilitating and cruel disease that leads to premature death but also makes profound economic sense as the cost of the incredible quantities of medicine needed every day, let alone the additional treatments including hospitalisation when severely ill is immense.

Please let us adopt this drugwithout delay, if indeed it is all it says it is.

nigel edward-few, chesham, bucks

Most of those commenting here seem to have completely missed the important point that only 5 - 15% of cases of any particular disease are caused by the kind of mutations which this development targets.

Ian Kemmish, Biggleswade, UK

BE CAREFUL

The mechanism stopping replication upon detection of a nonsense sequence is there for a reason.

The miracle treatment described in the article is highly likely to engender cancer and other malformations.

Yuu Takazaki, San Francisco, California, U.S.

Juxtaposed with the news that the former Pope is to be made a saint because he may have cured one person of Parkinson's disease, this happy news makes me wonder if the scientists involved will be likewise honoured. Probably they will be denigrated by some for trying to thwart the will of God.

E M Sedgwick, Eastleigh, Hampshire

While this is great, this is only a band-aid to the real problem. There's no guarantee that the drug will target the mutations, and it's not a magic drug that changes the entire genetic structure. A person would have to be on this crap for a lifetime. If we can affect mitosis that will ignore the jacked up DNA sequences, that would be one heck of a wonder drug...

James, Raleigh, NC

Richard, embryonic stem cell research IS legal. The debate is only whether or not federal funding is provided. The conditions that they could treat are often non-congenital, so it would be foolish to claim this discovery warrants abandoning stem cell research.

Jackie, Houston, TX

If true this is great news. As a CF sufferer aged 47 I have heard of lots of drugs and treatments that are going to cure CF but that have never materialised. e.g. gene therapy. Ten years ago we were told we would have a cure in five years and we are still waiting for it. The drug if it works will be good at halting the process of CF which is a progressive illness but will be able to do nothing for the damage that has already been done to the lungs, pancreas and liver. I just hope this is not more press hype.

Philip Hornsby, Scarborough, N.Yorkshire

I think this is fantastic and can't wait to hear more about it. I have three children with Sanfilippo Syndrome which is related to Hurler Syndrome.

www.caringbridge.org/ca/bennettboys

Alicia, Ione, CA

If it works in human beings, and can cure existing congenital disorders, previously considered incurable, then it will truly qualify for the description "miracle drug".

Colin Berry, Antibes, France

Good reason not to legalize dangerous embryonic stem cell research, which also kills an unborn human being.

Richard, Dubuque, USA/Iowa

Yes, I found the article in J Clin Pharmacol, published on April 2007, but not in NATURE as they say.

Zhaisxia, Ulm, Germany

God??????? What's the matter with people? If you believe in deities and that they create us (oh brother, are we still so unadvanced as a society?) it looks more like we're trying desperately to create chemicals that will fix what "they" mess up. Time to wake up to reality people. Oops, wait, that's what the opiate is for...

Cynthia, Juneau, Alaska

Hell! what a way to stay thin. Unfortunately you die in the process if someone's hand slips in production. But no matter the Democrats will dig up your name when it's time to vote for president and if you waant more socialism; especially while they follow Europe to ban all HEALTH PRODUCTS in our LAND/ What the Hell are you focusing on weight when there is so much else in this world to worry about. You get born, you grow up and old and die. What hapens inbetween is not anyone elses business but your own. Socialists always like to mess in other folks life.

john smith, Valencia, california

Is osteogenesis imperfecta one of the diseases this treatment could improve?

Mike Condra, Tracy, CA

Tremendous kudos to the researchers. Gene Therapy still has a mountain to climb. This drug at least creates a chair lift or two on this mountain.
www.thegenesherpa.blogspot.com

Steve Murphy MD, New York, NY, USA

Anybody hear if it is effective for Multiple Sclerosis?

God Bless all that this drug may help.

It is truly a miracle for those of us who are affected by chronic diseases.

Walt, LA,

I am very thankfull that this drug can have such widespread
effect. this is very good news for all of mankind.
Let us hope that such breakthroughs can be made in many more categories
of crippling and degenerative diseases

Larry P usateri, Bethel Park ,

Bravo to the scientists...thank you and God bless ya'll!

dan, gainesville, ga

im a hemophiliac and this is the best news ive heard in my entire life.

sheperd, los angeles, los angeles ,CA

I'm THRILLED! My wife has MD - and wow - what an annswer to our prayers.

matt, hillsboro, or

Awesome. I have worked with a kid with Duchenne, it is horrible, I hope this new therapy helps people out.

David Powell, Cape May , NJ, USA