Entries by Marie-Catherine Du Berger

Translarna™ (ataluren) is the First Therapy Approved in Brazil for DMD

– Ambulatory Duchenne patients who are five years and older with a nonsense mutation can now access a treatment that targets the underlying cause of DMD –   PTC Therapeutics, Inc. today announced that Translarna™ (ataluren) has been granted marketing approval from the Brazilian National Health Surveillance Agency (ANVISA) under rare diseases procedure, for the […]

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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 […]

CLINICAL TRIAL OF ATALUREN IN CANADA

PTC124-GD-041-DMD (Also known as study 041) The clinical trial of ataluren in nonsense mutation Duchenne muscular dystrophy (NMDMD) Study 041 is a new clinical trial for boys and young men who are living with nonsense mutation Duchenne Muscular Dystrophy (nmDMD). Who Can Enter Study 041? To ensure that participants who enroll in Study 041 get […]

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New drug for DMD being reviewed by the FDA

Sarepta Announces FDA Acceptance of Golodirsen (SRP-4053) New Drug Application for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 53 Press release here: Sarepta.com   Sarepta Therapeutics, Inc. announced the Food and Drug Administration, Division of Neurology had accepted its New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053) and provided a regulatory […]