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A young boy died in a trial for Pfizer’s experimental gene therapy for Duchenne muscular dystrophy, the company told patient advocates Tuesday. The boy was enrolled in Daylight, a trial studying the treatment in boys aged 2 or 3. The boy had received the therapy early last year, Pfizer told the advocates in a note posted online by Parent Project Muscular Dystrophy. Pfizer said it had not yet determined precisely what happened or whether it was linked to the treatment. Deaths and severe side effects linked to gene therapy have generally happened soon after dosing.

For a third time, Sarepta Therapeutics has convinced a top Food and Drug Administration official to overrule the prevailing view of their staff and approve a drug for Duchenne muscular dystrophy. On Thursday evening, the FDA announced it expanded the approval of Elevidys, Sarepta’s Duchenne gene therapy, to cover nearly all patients, regardless of age or wheelchair status, despite the fact that the drug failed a large, Phase 3 trial last year. That decision, documents concurrently released by the agency show, was made almost exclusively by Peter Marks, the agency’s director of the Center for Biologics Evaluation and Research. He...

Ulster University scientists have made a remarkable breakthrough which it is hoped could cure hereditary blindness. They have discovered a treatment that can target the gene that leads to corneal dystrophy—a condition that causes cloudy deposits on the cornea. […] Currently, those with corneal dystrophy face a lifetime of treatment and the only option for those who go blind is a corneal transplant from a donor eye, but this has risks and varying success rates. […] But the new treatment, developed by scientists at the University’s School of Biomedical Science, currently involves just one injection into the eye, which destroys...

Clone a sheep, and you are headline news. Publish a study detailing how you can treat the symptoms of muscular dystrophy using stem cells, and a significant point gets buried by the media — if that progress and those stem cells were not the result of politically correct and media-favored embryonic stem cell research. A study published online Wednesday by the scientific journal Nature reported the results of research led by Giullo Cossu, director of the stem cell institute at the San Raffaele Scientific Institute in Milan, Italy. Two golden retrievers severely disabled by muscular dystrophy were able to walk...

How would you like to be a regular citizen working at your job at home who gets a telephone call by a seriously husky voiced male directly saying....."this is the lockup, we have a warrant out for your arrest, (my name)."..... Takes one aback just a little, don't you think? Just needed to tell all FReepers ........... this is how the 'Muscular Dystrophy Association' chooses to call Americans in order to try to find donors here in my area. After thinking for a moment or two I told the guy on the phone to go f**k himself after he belatedly...

07/26/04 -- Researchers have found a delivery method for gene therapy that reaches all the voluntary muscles of a mouse - including heart, diaphragm and limbs ? and reverses the process of muscle-wasting found in muscular dystrophy. "We have a clear 'proof of principle' that it is possible to deliver new genes body-wide to all the striated muscles of an adult animal. Finding a delivery method for the whole body has been a major obstacle limiting the development of gene therapy for the muscular dystrophies. Our new work identifies for the first time a method where a new dystrophin gene...

Somewhere in Germany is a baby Superman, born in Berlin with bulging arm and leg muscles. Not yet 5, he can hold seven-pound weights with arms extended, something many adults cannot do. He has muscles twice the size of other kids his age and half their body fat. DNA testing showed why: The boy has a genetic mutation that boosts muscle growth. The discovery, reported in Thursday's New England Journal of Medicine, represents the first documented human case of such a mutation. Many scientists believe the find could eventually lead to drugs for treating people with muscular dystrophy and other...