January 3, 2019

Wave Life Sciences Duchenne Clinical Trial Selected for FDA Complex Innovative Trial Designs Pilot Program

Wave Life Sciences announced that the planned Phase 2/3 efficacy and safety trial for its lead Duchenne clinical program has been selected for the U.S. Food and Drug Administration (FDA) pilot program for complex innovative…

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January 3, 2019

Catabasis Phase 3 PolarisDMD Trial for Edasalonexent Update & Additional Trial Sites Open for Enrollment

Catabasis Pharmaceuticals shared an update on clinical trial sites in the United States and recent international clinical trial application approvals to perform the Phase 3 PolarisDMD trial for edasalonexent (CAT-1004) in Duchenne. PolarisDMD clinical trial…

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December 20, 2018

Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Golodirsen (SRP-4053)

PPMD is excited to learn that Sarepta Therapeutics, Inc.  announced that it has completed the submission of its rolling New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053), a phosphordiamidate morpholino oligimer engineered to treat…

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December 13, 2018

ICER Announces the Review of Duchenne Products – What Does This Mean?

Thanks to decades of advocacy, funding and innovation, our healthcare landscape is rapidly changing. As new therapies emerge into the marketplace, patients, providers, and payers are faced with challenges as to how to discern which…

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December 6, 2018

Wave Life Sciences Announces Positive Phase 1 Results for WVE-210201 in Duchenne Muscular Dystrophy

  Wave Life Sciences announced positive safety and tolerability results from the WVE-210201 Phase 1 clinical trial today. PPMD continues to be optimistic about the progress the team at Wave Life Sciences is making with…

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November 20, 2018

Santhera Enters into Agreement to Acquire Option for Exclusive Sub-License of Vamorolone

PPMD has learned that Santhera Pharmaceuticals and Idorsia Ltd have entered into an agreement under which Santhera will acquire the option to exclusively in-license vamorolone, currently being developed by Reveragen. Idorsia currently holds the license…

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November 15, 2018

WATCH: PolarisDMD – Phase 3 Clinical Trial of Edasalonexent – Webinar Recording

PPMD and Catabasis as hosted a webinar on Wednesday, November 7 to discuss the Phase 3 PolarisDMD clinical trial studying edasalonexent in Duchenne muscular dystrophy, which is enrolling boys ages 4 to 7 (up to…

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November 13, 2018

Solid Provides Update on IGNITE DMD Clinical Trial

  Solid Biosciences Inc. today shared on a updated on the IGNITE DMD clinical trial, noting the dosing of two additional patients with SGT-001 gene transfer and the company’s plan to report preliminary results from IGNITE…

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October 15, 2018

WATCH: Is Myostatin Still a Potential Therapeutic Target? – Webinar Recording

Myostatin is a negative regulator of muscle growth – a protein that stops muscle building and doesn’t let them get too big, as part of the natural regeneration process. In other words, if we can…

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October 5, 2018

NS Pharma Announces Positive Results of US/Canada Phase II Clinical Trial of Viltolarsen at World Muscle Society

NS Pharma announced positive results of a US/Canada Phase II clinical trial of viltolarsen at the World Muscle Society meeting held in Mendoza, Argentina. The preliminary findings suggest clinical improvement as measured by timed function…

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