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Amber Sapp was browsing the Internet late one night in August when she happened to find out that her 12-year-old son’s clinical trial had failed.
Every four weeks for two-and-a-half years, she had shuttled Garrett to a hospital nearly six hours away. There, he was prodded and pricked with needles in the hope that the antibody treatment being tested would reverse a devastating genetic disease called Duchenne muscular dystrophy. But an early data analysis, Sapp learnt, had shown that the treatment wasn’t working.
The thought of wasting Garrett’s limited time with a failed trial was hard enough. The news was all the more disturbing because it didn’t come from the trial organizers, but through a Facebook post from another parent. “It was upsetting that we found out that way,” says Amber. “It sent everybody on Facebook into a tizzy.” Even Garrett’s local clinical-trial coordinator, someone who should have had intimate knowledge of what was happening with the research, hadn’t yet heard the news.
Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystr...The proposed phase I clinical trial is a...
Concordant but varied phenotypes among Duchenne muscular dystrophy patient-specific myoblasts derived using a human ...Duchenne muscular dystrophy (DMD) remain...
MSU students develop exoskeleton app for patient with Duchenne muscular dystrophyZach Smith has Duchenne muscular dystrop...
Joline Dalton, MS, CGCJoline Dalton is a board certified genet...
Kay Davies, CBE, DBE, FMedSci, FRSDr. Kay Davies is elected Dr. Lee’s Pr...
Study of an Investigational Drug, BMS-986089, in Ambulatory Boys With DMDThe purpose of this study is to determin...