Category Archives: Muscular Dystrophy Treatment

Pictured: Randomized data over a strand of DNA/Nicole Bean for BioSpace Rare diseases, by their very nature, dont fit the moldso neither should the trials for therapies designed to treat them nor the regulatory process to approve them. This was the tone set during a recent panel discussion where Peter Marks, director of the FDAs Center for Biologics Evaluation and Research, argued that non-randomized, single-arm trials could be the best option when testing certain gene therapies for rare diseases Continue reading →

Applied Clinical Trials: What sets Vamorolone apart from other therapies in this market? Sharon Hesterlee: Vamorolone is a little bit different because we've heard a lot about gene therapies and some pretty high tech, very specific, genetically focused medicines in the Duchenne space Continue reading →

HuidaGene Therapeutics investigational gene-editing therapy, called HG302, for Duchenne muscular dystrophy (DMD) has been granted a rare pediatric drug designation by the U.S. Food and Drug Administration (FDA). Continue reading →

Although the road has not been smooth, 2023 was witness to unprecedented progress in the treatment landscape of Duchenne muscular dystrophy (DMD), with the approval of Sarepta Therapeutics delandistrogene moxeparvovec (SRP-9001; Elevidys) earlier this summer.1 Despite the milestone approval, only a small percentage of patients with DMD currently stand to benefit from the therapy. Continue reading →

'I was so lucky!' Gilbert Gottfried's widow pays tribute to Aladdin star one year on from his death   Yakima Herald-Republic Continue reading →

Going beyond customs: The Hindu Editorial on customs exemption for drugs, food imported for treatment of rare diseases   The Hindu Continue reading →

Entrada Therapeutics Appoints Dr. Bernhardt Zeiher to its Board of Directors   Marketscreener.com Continue reading →

What is Duchenne Muscular Dystrophy? Continue reading →

28 Reasons we love Rochester one for every day of the Month of Love   Rochester City Newspaper Continue reading →

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