October 4, 2023

REGENXBIO announces interim clinical data from Phase I/II AFFINITY DUCHENNE™ trial of RGX-202

REGENXBIO Inc. has announced additional interim safety data and initial efficacy data from the Phase I/II AFFINITY DUCHENNE™ trial of RGX-202, an investigational gene therapy product for the potential treatment of Duchenne. RGX-202 utilizes a…

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September 26, 2023

Edgewise Therapeutics Now Enrolling GRAND CANYON Study of EDG-5506 in Individuals with Becker Muscular Dystrophy

Edgewise Therapeutics has announced the start of enrollment of GRAND CANYON, the company’s global pivotal study of EDG-5506 in individuals with Becker. GRAND CANYON is an expansion of the CANYON study, which was over-enrolled. EDG-5506…

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September 21, 2023

Entrada Therapeutics starts dosing in ENTR-601-44 Phase 1 trial

Entrada Therapeutics has announced that the first participant has been dosed in its Phase 1 clinical trial evaluating ENTR-601-44, the company’s proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO), for the potential treatment of…

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September 15, 2023

PTC Therapeutics Announces Negative European Medicines Agency Opinion Regarding Full Authorization of Translarna

PTC Therapeutics today shared that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended not renewing the Marketing Authorization for Translarna™ (ataluren), a treatment for Duchenne patients…

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August 30, 2023

FibroGen Announces Topline Data from Phase 3 LELANTOS-2 Study

FibroGen has announced topline results of the Phase 3 LELANTOS-2 trial of pamrevlumab, their investigational anti-fibrotic monoclonal antibody, for ambulatory patients with Duchenne muscular dystrophy.  Although generally safe and well tolerated, pamrevlumab did not meet…

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June 29, 2023

Italfarmaco Group Announces NDA Accepted for Givinostat

PPMD is excited to learn that the FDA completed its filing review and accepted Italfarmaco Group’s New Drug Application (NDA) for Givinostat, the company’s proprietary histone deacetylase (HDAC) inhibitor for the treatment of individuals with…

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June 27, 2023

Edgewise Therapeutics announces positive topline 12-month data from EDG-5506 ARCH study

Edgewise Therapeutics today announced topline 12-month data from the ARCH study of EDG-5506, an orally administered small molecule designed to prevent contraction-induced muscle damage in patients with Becker muscular dystrophy. The ARCH study is evaluating…

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June 21, 2023

Santhera announces exclusive North America license agreement with Catalyst Pharmaceuticals for vamorolone

PPMD has learned that Santhera Pharmaceuticals has entered into an agreement granting exclusive North America license for vamorolone to Catalyst Pharmaceuticals. Santhera will continue to keep commercialization rights in Europe, while Catalyst will obtain commercialization…

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June 21, 2023

WATCH: ENVISION Study Update With Sarepta Therapeutics (Webinar Recording)

Last week Sarepta Therapeutics joined PPMD to discuss ENVISION, their upcoming 18-month placebo-controlled Phase 3 study of SRP-9001 (delandistrogene moxeparvovec) for older ambulatory and non-ambulatory individuals with Duchenne. Matthew Furgerson, PhD, Medical Director, Clinical Development…

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June 15, 2023

PPMD, CureDuchenne, Muscular Dystrophy Association Announce Collaborative Project to Focus on Re-Dosing Gene Therapy in Duchenne Muscular Dystrophy

CureDuchenne, Muscular Dystrophy Association (MDA), and Parent Project Muscular Dystrophy (PPMD) today announced a collaborative clinical trial grant to test repurposing of the FDA-approved drug Vyvgart (developed by argenx), for its potential ability to reduce…

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