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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February 12, 2019

WATCH: Vamorolone Clinical Trials in Duchenne – Webinar Recording

On January 30, 2019, ReveraGen joined Parent Project Muscular Dystrophy for a webinar to provide the community with an overview of the vamorolone drug development program for Duchenne, including results showing improved muscle function in…

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February 8, 2019

How Do I Find a Clinical Trial That’s Right for Me?

Are you getting confused by all the news and social media posts about clinical trials? Not sure which trials or studies may be appropriate for you or your child? You are not alone! Even healthcare…

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February 7, 2019

Solid Biosciences Announces Intention to Dose Escalate in IGNITE DMD Clinical Trial

Solid Biosciences announced today plans to move to a higher dose of SGT-001 in IGNITE DMD, their Phase 1/2 clinical trial evaluating SGT-001 microdystrophin gene therapy as a potential treatment for Duchenne. This decision is…

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February 6, 2019

NS Pharma Begins Rolling NDA Submission to FDA for Viltolarsen (NS-065/NCNP-01)

PPMD is pleased to learn that NS Pharma has initiated submission of a rolling New Drug Application (NDA) seeking accelerated approval for viltolarsen (NS-065/NCNP-01), an investigational drug for the treatment of Duchenne in patients amenable…

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January 25, 2019

WATCH: Translarna® (ataluren) Development Update – Webinar Recording

On January 16, 2019, PTC Therapeutics joined Parent Project Muscular Dystrophy for a webinar update to provide the community with an overview of the Translarna development program with a focus on clinical trials that are…

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January 22, 2019

Capricor Announces Positive Outcomes from Comprehensive Multidisciplinary Meeting with FDA

Capricor Therapeutics announced that it had positive outcomes in its comprehensive multidisciplinary meeting with the FDA regarding development of the company’s novel cell therapy candidate, CAP-1002, after being placed on voluntary hold last month. To…

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