September 28, 2020

Sarepta Reports Sustained Functional Improvement Two Years After Treatment with SRP-9001 Gene Therapy

PPMD is excited to share today’s announcement from Sarepta Therapeutics regarding two-year follow up results from four clinical trial participants who received SRP-9001 (AAVrh74.MHCK7.micro-dystrophin). Results presented at the Virtual 25th International Congress of the World…

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September 24, 2020

WATCH: Resiliency in Results – What We Learn When Trials Are Terminated (Webinar Recording)

No other community knows the importance of resiliency like the Duchenne community, as highlighted by our communal commitment to ensure that all people living with Duchenne have access to safe, accessible therapies. Clinical trials are…

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September 23, 2020

Santhera Announces Publication of Long-Term Clinical Data with Vamorolone in Patients with Duchenne

Santhera Announces Publication of Long-Term Clinical Data with Vamorolone in Patients with Duchenne Santhera announced a new publication in the journal PLOS Medicine describing new open-label, long-term clinical data on the safety, tolerability and efficacy…

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September 21, 2020

Pfizer Shares Update on PF-06939926 Gene Therapy Trial During Company’s Virtual Investor Day

Pfizer recently shared an update on their mini-dystrophin replacement gene therapy product PF-06939926 during their virtual Investor Day. In total 18 boys have received the gene therapy product as part of the Phase 1b study….

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September 18, 2020

Catabasis Shares September 2020 Newsletter

Catabasis Pharmaceuticals has shared the latest edition of their community newsletter.  Their plan is to share top-line results from the Phase 3 PolarisDMD trial of edasalonexent with the Duchenne community later this year. As highlighted in…

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September 14, 2020

cTAP Study Shows Real-World, Natural History Data Comparable To Placebo Data From Trials in Duchenne

The Collaborative Trajectory Analysis Project (cTAP) announced results of a study looking at real world data and natural history data and comparing it to placebo arm data from past clinical trials. The goal of this…

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September 14, 2020

Resilience Is Strength: Thank You for Fueling Our Fight to End Duchenne

As we celebrate the resiliency of our community this week as part of Duchenne Action Month, I have been reflecting upon all that we continue to accomplish, even in this time that is anything but…

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September 11, 2020

Santhera Announces Full Enrollment of ReveraGen’s VISION-DMD Study with Vamorolone in Duchenne

Santhera announced that ReveraGen’s VISION-DMD study for vamorolone in Duchenne has completed enrollment. Subject to a positive study outcome, this could allow for a regulatory submission to the FDA in the fourth quarter of 2021….

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September 10, 2020

Sarepta Therapeutics Provides Program Update for SRP-9001 Micro-Dystrophin Gene Therapy

Sarepta Therapeutics announced that it has completed a meeting (Type C) with FDA’s Office of Tissues and Advanced Therapies (OTAT), an office that is part of the Center for Biologics Evaluation and Research (CBER). The…

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September 2, 2020

WATCH: NS Pharma Announcement of VILTEPSO (viltolarsen) – Webinar Recording

NS Pharma joined PPMD for a webinar on August 26, 2020 to provide an update about VILTEPSO™ from Ben Yungher, PhD, NS Pharma’s Medical Science Liaison, and its associated patient support program from Randy McGonigal,…

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