October 27, 2025

Avidity Biosciences to be Acquired by Novartis

Yesterday, Avidity Biosciences announced a definitive agreement to be acquired by Novartis. The transaction includes Avidity’s late-stage programs, including delpacibart zotadirsen (del-zota) for Duchenne. According to Avidity’s announcement, the company and Novartis intend to keep…

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October 24, 2025

Italfarmaco Addresses Patient Deaths, DUVYZAT Safety Profile in Community Letter

Italfarmaco, the company that developed DUVYZAT® (givinostat), has shared a letter to the Duchenne muscular dystrophy community addressing concerns raised in recent online, community-based discussions about patient deaths involving individuals living with Duchenne who were…

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October 17, 2025

Newly Published Study Evaluates Patients’ and Caregivers’ Acceptable Risk from Gene Therapy

A new threshold study, “Re-evaluating Acceptable Risk of Death from Gene Therapy,” has been published in the European Journal of Medical Genetics. This work addresses a critical question: how should risk and benefit be assessed…

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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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