October 14, 2025

Avidity Biosciences Announces Positive Pre-BLA Meeting with FDA for del-zota

Avidity Biosciences, Inc. has announced the completion of a positive pre-Biologics License Application (BLA) meeting with the U.S. Food and Drug Administration (FDA) regarding its upcoming BLA submission of delpacibart zotadirsen (del-zota) for the treatment…

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September 10, 2025

Avidity Biosciences Announces Positive New Data from EXPLORE44® and EXPLORE44-OLE™ Phase 1/2 Studies

Today, Avidity Biosciences announced new data from its EXPLORE44® and EXPLORE44-OLE™ Phase 1/2 studies of del-zota in people living with Duchenne muscular dystrophy amenable to exon 44 skipping. Del-zota is designed to deliver phosphorodiamidate morpholino…

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August 13, 2025

Solid Biosciences Shares Update on INSPIRE DUCHENNE Trial of SGT-003

Solid Biosciences has shared an update on the company’s Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003 for the treatment of individuals living with Duchenne. SGT-003 is a gene therapy candidate for the treatment of Duchenne…

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August 4, 2025

Dyne Therapeutics, Inc. announced today FDA Breakthrough Therapy Designation for DYNE-251 in Duchenne Muscular Dystrophy

Dyne Therapeutics has announced FDA Breakthrough Therapy Designation for DYNE-251 in Duchenne Muscular Dystrophy. This designation reflects encouraging early results from the DELIVER clinical trial, where patients demonstrated sustained functional improvements, such as faster time…

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July 23, 2025

FDA Grants Breakthrough Therapy Designation to Avidity’s del-zota

Avidity Biosciences, Inc. today shared that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to del-zota (AOC 1044) for the treatment of Duchenne in individuals amenable to exon 44 skipping.  Del-zota…

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July 22, 2025

Update on ELEVIDYS Shipments in the U.S.

This evening, Sarepta Therapeutics issued a press release stating that it has voluntarily and temporarily paused all shipments of ELEVIDYS for Duchenne in the United States, effective close of business tomorrow, July 22, 2025. According…

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July 18, 2025

ELEVIDYS Update

Updated July 18, 2025 at 8:35 PM ET Sarepta Therapeutics has provided an update indicating that the company has received an informal request from the U.S. Food and Drug Administration (FDA) to voluntarily halt shipment…

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July 17, 2025

Sarepta Therapeutics Announces Update on ELEVIDYS Label, Company Restructuring

Yesterday afternoon, Sarepta issued a press release and community letter which included updates related to the non-ambulatory label for ELEVIDYS. While ELEVIDYS continues to be available for individuals living with Duchenne who are ambulant, Sarepta…

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July 11, 2025

Capricor Therapeutics Receives Complete Response Letter Regarding Deramiocel (CAP-1002)

On July 9th, the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for Capricor’s Biologics License Application (BLA) for Deramiocel (CAP-1002), an investigational cell therapy for Duchenne cardiomyopathy. This decision means…

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July 2, 2025

Webinar Recording: Edgewise Therapeutics – Update on Sevasemten Program in Becker and Duchenne

Edgewise Therapeutics recently joined PPMD for a community webinar for an update on the company’s sevasemten program in Duchenne and Becker. The Edgewise team reviewed new Open-label Extension MESA data in Becker, as well as…

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