January 25, 2022

Capricor & Nippon Shinyaku Enter Partnership for Commercialization and Distribution of CAP-1002 in the U.S.

Capricor Therapeutics and Nippon Shinyaku (US subsidiary: NS Pharma) have announced that they have entered into an agreement for the exclusive commercialization and distribution in the United States of CAP-1002, Capricor’s novel cell therapy candidate…

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January 24, 2022

Thanking PPMD’s Kathi Kinnett for 20+ Years of Service to the Duchenne Community as She Retires

PPMD celebrates Kathi Kinnett, MSN, APRN, a dedicated member of our incredible Care Team for 10 years, as she announces her retirement at the end of this month. Kathi joined PPMD in 2012 after years of…

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January 21, 2022

PPMD’s Adult Advisory Committee (PAAC) in Action! 2021 Highlights & Looking Ahead

2021 was a busy and productive year for the PPMD Adult Advisory Committee (PAAC), PPMD’s advisory group composed of adult members of our Duchenne and Becker community. The PAAC successfully participated in local and federal…

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January 20, 2022

Dyne Therapeutics Announces FDA Clinical Hold on IND Application for DYNE-251 in Duchenne

Dyne Therapeutics recently shared that the FDA issued a clinical hold on their Investigational New Drug (IND) application for the Phase I/II study of their exon skipping therapy utilizing a conjugated monoclonal antibody DYNE-251. For…

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January 13, 2022

Solid Provides Updates on IGNITE DMD Study and SGT-003 Pre-Clinical Data

Solid Biosciences has shared an update from IGNITE DMD, an ongoing Phase I/II clinical trial evaluating its gene therapy candidate SGT-001 in participants with Duchenne, as well as developments for the company’s next generation gene…

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January 11, 2022

Sarepta Announces Topline Results from Part 2 of Study SRP-9001-102 Evaluating Duchenne Gene Therapy Candidate

Sarepta Therapeutics has shared an update from Part 2 of Study SRP-9001-102, an ongoing clinical trial evaluating Sarepta’s gene therapy candidate SRP-9001 in participants with Duchenne. SRP-9001 is an investigational gene transfer therapy intended to…

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January 6, 2022

REGENXBIO Announces FDA Clearance of IND for Clinical Trial of RGX-202 Gene Therapy Candidate for Duchenne

PPMD is excited to share that REGENXBIO has announced that they have received clearance from the FDA on their Investigational New Drug (IND) application for RGX-202, a micro-dystrophin gene therapy product. With this clearance REGENXBIO…

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January 5, 2022

Edgewise Therapeutics Announces Positive Topline Results From EDG-5506 Phase 1b Trial in Adults With Becker

Edgewise Therapeutics today announced positive topline results from the Phase 1b clinical trial of EDG-5506 in Becker muscular dystrophy. EDG-5506 is an oral small molecule muscle stabilizer which seeks to protect the muscle from injuries…

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January 5, 2022

Sarepta Therapeutics Shares MOMENTUM, Study SRP-5051-201, Update

Sarepta Therapeutics has shared an update that SRP-5051-201, the MOMENTUM study (NCT04004065), is now recruiting Part B. MOMENTUM is a Phase 2, two-part, multiple-ascending-dose study of SRP-5051 for dose determination, then dose expansion, in patients…

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January 4, 2022

Thank YOU for Joining the Power of Us!

I am grateful for your generosity this year and excited to announce that we have reached our goal of providing supplemental funding to the Wellstone Muscular Dystrophy Cooperative Research Centers. The Power of Us is…

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