Free Republic

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...

Researchers have discovered that an existing cancer drug could have potential as a treatment for muscular dystrophy. The researchers found that the drug—known as a colony-stimulating factor 1 receptor (CSF1R) inhibitor—helped slow the progress of Duchenne muscular dystrophy in mice by increasing the resiliency of muscle fibers. "This is a class of drug that is already being used in clinical trials to treat rare forms of cancer," says Dr. Farshad Babaeijandaghi. Duchenne muscular dystrophy (DMD) is a severe genetic disorder that leads to progressive muscle weakness and degeneration. "While this is not a cure, it could significantly delay disease progression,...

CureDuchenne will sponsor a one-year research program that will validate Dr. Giulio Cossu’s stem cell research under GMP (Good Manufacturing Procedures) conditions. Dr. Cossu’s long-term objective is to conduct phase I/II clinical trials in Duchenne muscular dystrophy (“DMD”) pediatric patients with this novel cell therapy protocol based on intra-arterial delivery of donor mesoangioblasts. The immediate objective of Dr. Cossu is to develop a clinical grade protocol that will allow transplantation of patients with mesoangioblasts in a successive clinical trial. During this study, Dr. Cossu will prepare an IMPD, the European equivalent of a US IND (Investigational New Drug Application),...

SYLACAUGA — His name is Dwayne, he's 16 and he is dying of Duchenne Muscular Dystrophy, the type of muscular dystrophy that destroys its victims early in life. Dwayne's mother, Julie Crouch of Sylacauga, said she noticed when her son was 3-years-old there was something wrong with the way he walked. She took him to a doctor and he was diagnosed with MD. Dwayne has been confined to a wheelchair since age 8. The happy, young fellow is quick to tell you he is not going to die. He insists he is going to live and take care of his...