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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December 20, 2021

Update on Pfizer’s Phase 1B Open-Label Mini-Dystrophin Gene Therapy Trial for Duchenne

Today we learned that there has been a devastating loss of a young man that was participating in Pfizer’s Phase 1B Open-Label study. This study is evaluating PF- 06939926 which is Pfizer’s AAV9 Gene Therapy…

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December 17, 2021

Gene Editing Therapy Start-up Secures $3.4 Million Grant for Duchenne Research

MyoGene Bio has received a $3.4 million grant from the California Institute of Regenerative Medicine to advance its pre-clinical gene-editing studies in Duchenne, thanks to support from Parent Project Muscular Dystrophy (PPMD), Duchenne UK, and…

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December 15, 2021

Second Chance — Win a NEW Nintendo Switch OLED!

THANK YOU, Duchenne Registry participants! As you might have heard, The Duchenne Registry was thrilled to recently give away a Nintendo Switch OLED to a Duchenne Registry participant who completed all of their surveys. The…

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December 15, 2021

Edgewise Initiates ARCH Follow-On Open Label Becker Study; Will Report Phase 1b Topline Results on January 5

Edgewise Therapeutics has announced the initiation of the ARCH open label study of EDG-5506 in Becker muscular dystrophy, which will enroll participants from its Phase 1b clinical trial as well as new participants with Becker….

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December 6, 2021

PPMD’s Drug Development Roundtable Meets to Explore Utilization of External Controls to Accelerate Research

At the heart of PPMD’s mission to end Duchenne lies our unwavering commitment to accelerate research. As part of our comprehensive research strategy, this includes a focus on enhancing the drug development process to ensure…

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