November 19, 2025

Avidity Biosciences Announces U.S. Managed Access Program for del-zota

Avidity Biosciences, Inc. has announced its Managed Access Program (MAP) for delpacibart zotadirsen (del-zota) for eligible individuals living with Duchenne who are amenable to exon 44 skipping in the United States. MAPs allow a limited…

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November 14, 2025

FDA Approves New Safety Warning and Revised Indication for ELEVIDYS

Today the FDA announced a significant update to the labeling and indication of ELEVIDYS for the treatment of individuals living with Duchenne following reports of death based on acute liver failure (ALF) in non-ambulatory patients…

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November 5, 2025

Solid Biosciences Shares Interim Data from INSPIRE DUCHENNE Trial of SGT-003

Solid Biosciences Inc. has announced positive new interim data from the Phase 1/2 INSPIRE DUCHENNE clinical trial and provided an update on its planned meeting with the U.S. Food and Drug Administration (FDA) to discuss…

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November 4, 2025

Sarepta Announces Completion of ESSENCE Trial for Exon 45 & 53 Skipping Therapies

Sarepta Therapeutics has announced top-line results from the ESSENCE trial, a global, randomized double-blind, placebo-controlled Phase 3 confirmatory study evaluating the efficacy and safety of AMONDYS 45 (casimersen) and VYONDYS 53 (golodirsen) in individuals living…

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November 4, 2025

PPMD Provides $400,000 in Funding to MyoGene Bio Through PPMD Venture Pathways Program to Support Development of Gene Editing Platform

PPMD and MyoGene Bio (MyoGene) are excited to announce that PPMD has provided $400,000 in funding to MyoGene through PPMD Venture Pathways, the organization’s venture-philanthropy initiative that provides industry funding to accelerate therapeutic development for…

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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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