January 11, 2024

Introducing PPMD’s Gene Therapy Hub

We are excited to announce the launch of PPMD’s Gene Therapy Hub! Gene therapy has made significant strides in Duchenne, with one therapy approved and other potential therapies in various stages of development. As our…

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January 8, 2024

PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51

PepGen has announced that the first participant has been dosed in its Phase 2 CONNECT1-EDO51 study, which will evaluate the safety and tolerability of PGN-EDO51, the company’s next-generation conjugated-PMO exon skipping therapy for the treatment…

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January 3, 2024

Dyne Therapeutics Announces Positive Initial Clinical Data from DELIVER Trial in Duchenne Patients

Dyne Therapeutics today announced positive initial clinical data from its DELIVER trial of DYNE-251 in patients with Duchenne who are amenable to exon 51 skipping. Dyne-251 is an exon skipping product that combines a PMO…

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December 22, 2023

Sarepta Therapeutics Submits Efficacy Supplement to Expand the ELEVIDYS Label

Sarepta Therapeutics, Inc. today announced the submission of an efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS to expand its labeled indication. The company also shared that it completed the Phase 3 EMBARK…

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December 21, 2023

PPMD & Community Partners Urge Timely, Transparent FDA Review of EMBARK Data

Today, PPMD joined the Best Day Ever Foundation, CureDuchenne, the Jett Foundation, the Little Hercules Foundation, The Muscular Dystrophy Family Foundation, and Team Joseph in sending a letter to the Federal Drug Administration (FDA) regarding…

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December 15, 2023

Wave Life Sciences Announces Initiation of Dosing in Phase 2 Duchenne Clinical Trial of WVE-N531

Wave Life Sciences recently shared that the company has initiated dosing and has fully enrolled the Phase 2 FORWARD-53 trial of investigational WVE-N531, a potential exon skipping therapy targeting people with Duchenne amenable to exon…

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December 12, 2023

Watch: Community Update on the New INSPIRE DUCHENNE Clinical Study of SGT-003 (Webinar Recording)

Solid Biosciences recently joined PPMD for a community webinar to provide an overview of INSPIRE DUCHENNE, a first-in-human clinical study evaluating SGT-003, a next-generation microdystrophin gene therapy candidate for the treatment of Duchenne. The Solid…

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December 5, 2023

PTC Therapeutics Shares Update on Translarna™ Regulatory Activities

PTC Therapeutics, Inc. has shared an update on regulatory activities in Europe and the United States for Translarna™ (ataluren), a treatment for Duchenne patients with nonsense variants.  Europe: European Medicines Agency (EMA) Update Following the…

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November 29, 2023

Italfarmaco Announces PDUFA Extension for the FDA’s Review of Givinostat

Italfarmaco Group announced an extension in the FDA’s review of the New Drug Application (NDA) for Givinostat, the company’s proprietary histone deacetylase (HDAC) inhibitor for the potential treatment of Duchenne. The new Prescription Drug User…

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November 14, 2023

Solid Biosciences Receives FDA Clearance for Investigational New Drug Application for SGT-003 Gene Therapy Candidate

PPMD is pleased to learn that Solid Biosciences has received clearance by the U.S. Food and Drug Administration (FDA) for an Investigational New Drug (IND) application for SGT-003, the company’s next generation microdystrophin gene therapy…

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