November 25, 2025

Sarepta Announces FDA Approval to Begin ENDEAVOR Cohort 8 Dosing in Non-Ambulatory Individuals Living with Duchenne

Sarepta Therapeutics, Inc. has shared that the U.S. Food and Drug Administration (FDA) has approved dosing in Cohort 8 of ENDEAVOR, an open-label, Phase 1b study assessing the expression and safety of ELEVIDYS in multiple…

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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

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 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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August 27, 2025

Newly Published Consensus Guidelines Aim to Ensure Safe and Equitable Delivery of Gene Therapy in Duchenne

PPMD, in collaboration with the Muscular Dystrophy Association (MDA), is proud to share the release of newly published consensus guidelines for the delivery and monitoring of gene therapy in Duchenne muscular dystrophy (Duchenne). Developed in…

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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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July 29, 2025

Sarepta to Resume ELEVIDYS Shipments to Ambulatory Patients After FDA Recommends Removal of Voluntary Hold for Ambulatory Population

The U.S. Food and Drug Administration (FDA) has announced that the agency is now recommending the removal of Sarepta Therapeutics’ voluntary hold for ambulatory patients eligible to receive ELEVIDYS. In its own press release, Sarepta…

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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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