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FibroGen receives orphan drug designation for Pamrevlumab

FibroGen Receives Orphan Drug Designation from the U.S. FDA For Pamrevlumab for the Treatment of Duchenne Muscular Dystrophy   FibroGen, Inc. a leading biopharmaceutical company discovering and developing a pipeline of first-in-class therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the company’s anti-CTGF antibody, pamrevlumab, for […]

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New gene therapy for Duchenne muscular dystrophy

Audentes Therapeutics, Inc., a leading Adeno-associated virus (AAV)* based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular diseases; announced it had expanded its scientific platform and pipeline to advance vectorized antisense treatments for the treatment of Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). The Gene Therapy […]

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Phase 3 PolarisDMD trial of edasalonexent is now open for enrollment in Canada

Catabasis is enrolling boys ages 4 to 7 (up to 8th birthday), any mutation type, who have not been on steroids for at least the past six months.   What is Edasalonexent? Edasalonexent (CAT-1004 is being developed as a potential foundational disease-modifying therapy for all patients affected by DMD, regardless of their underlying mutation. It […]

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Sarepta Therapeutics announces positive results from the ESSENCE study

Sarepta Therapeutics, Inc., a leader in precision genetic medicine for rare diseases, announced positive results from its interim analysis of muscle biopsy endpoints comparing casimersen treatment to placebo in the ESSENCE study. ESSENCE is a global, randomized double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of casimersen and golodirsen in patients amenable to […]

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Idebenone for Duchenne muscular dystrophy

February 25, 2019 Santhera Pharmaceuticals announces results from the SYROS study.   The primary objective of this study was to evaluate the long-term evolution of the respiratory function in patients who maintained treatment with idebenone for up to 6 years compared to their preceding off-idebenone period.   Respiratory Function in DMD In boys and men […]

CRISPR & Duchenne muscular dystrophy

The success of CRISPR in a model of Duchenne muscular dystrophy Original story from Duke University Researchers at Duke University have shown that by using genome editing technology, CRISPR can safely and stably correct a genetic condition such as Duchenne Muscular Dystrophy (DMD). The study appears in the journal Nature Medicine. Links here   CRISPR/Cas9 […]