April 15, 2019

FibroGen Receives U.S. FDA Orphan Drug Designation for Pamrevlumab for the Treatment of Duchenne

FibroGen announced that FDA has granted Orphan Drug Designation for the company’s anti-CTGF antibody, pamrevlumab, for the treatment of people with Duchenne. According to their press release, all 21 non-ambulatory Duchenne patients in their ongoing…

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April 8, 2019

Audentes Therapeutics Announces Expansion of AAV Technology Platform and Pipeline for Duchenne

Audentes Therapeutics announced that it is expanding its scientific platform and beginning development of new AAV-based genetic medicines for Duchenne. Audentes is a leading AAV-based genetic medicines company based in San Francisco. The company is…

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April 5, 2019

Catabasis Shares March 2019 Newsletters

Catabasis shared the latest edition of their community newsletter (also available in Spanish), including an update on their currently recruiting Phase 3 PolarisDMD trial for edasalonexent in Duchenne. Download the Catabasis newsletters: English Spanish

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April 3, 2019

WATCH: Italfarmaco Provides Givinostat Update – Webinar Recording

On Wednesday, March 27, 2019, Italfarmaco joined Parent Project Muscular Dystrophy for a webinar to update the community about Italfarmaco’s development of Givinostat and currently enrolling Phase 3 confirmatory study in Duchenne muscular dystrophy. If…

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March 28, 2019

Sarepta Announces Positive Expression Results from the Casimersen (SRP-4045) Arm of ESSENCE Study

Sarepta Therapeutics announced positive results from its interim analysis of muscle biopsies at week 48, comparing casimersen treatment to placebo in the ESSENCE study, also known as study 4045-301. ESSENCE is a global, randomized double-blind,…

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March 25, 2019

Sarepta Provides Micro-dystrophin Gene Therapy Update

Sarepta Therapeutics hosted a webcast earlier this morning, providing an update on encouraging 9-month functional and creatine kinase (CK) data from baseline for the original four patients in the Phase 1 open-label study of Sarepta’s…

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February 27, 2019

Roche/Genentech General Program Update Letter to the Duchenne Community

As Roche/Genentech continues to advance its Phase 2/3 clinical development program for RG6206, an adnectin fusion protein designed to inhibit myostatin and stimulate muscle growth in ambulatory people with Duchenne, the company has provided an…

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February 25, 2019

Santhera’s SYROS Study Shows Long-term Efficacy with Idebenone in Slowing Respiratory Function Loss

Santhera Pharmaceuticals announced positive results from the SYROS study demonstrating that long-term treatment with idebenone consistently reduced the rate of respiratory function loss in patients with Duchenne for up to 6 years in a real-world…

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February 19, 2019

Catabasis Presents Data Supporting Edasalonexent as a Potential Foundational Treatment for Duchenne

Catabasis Pharmaceuticals, Inc. shared additional clinical results from the MoveDMD trial of edasalonexent at the XVII International Conference on Duchenne and Becker Muscular Dystrophy in Rome, Italy this weekend. We are pleased to learn that in…

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February 14, 2019

Sarepta Announces FDA Acceptance of Golodirsen NDA for People with Duchenne Amenable to Exon 53 Skipping

Sarepta Therapeutics, Inc. announced the FDA has accepted its New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053) and provided a regulatory action date of August 19, 2019. Golodirsen is a potential exon skipping…

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