News

January 14, 2021January 14, 2021

Dosing Begins in Phase 3 Trial of Pfizer’s Gene Therapy for Duchenne

News

The first boy has been dosed in a Phase 3 trial testing Pfizer‘s gene therapy, PF-06939926, in treating Duchenne muscular dystrophy (DMD). “The initiation of our pivotal trial, which is the ... Read more

January 12, 2021January 12, 2021

DMD Boys Experience Biological, Functional Gains in SRP-9001 Study

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The investigational gene therapy SRP-9001 safely leads to the production of a functional form of dystrophin in boys with Duchenne muscular dystrophy (DMD) — and to improvements in their ... Read more

January 11, 2021January 11, 2021

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

News, syndicated

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD ... Read more

January 7, 2021January 7, 2021

Pivotal Trial Begins for AMO-02 in Steinert Disease

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AMO Pharma has announced the initiation of REACH-CDM, a pivotal Phase 2/3 clinical trial that will assess the efficacy and safety of the investigational medication AMO-02 (tideglusib) in children and ... Read more

January 5, 2021January 5, 2021

Edgewise Will Use $95M Funding to Advance EDG-5506 for BMD, DMD

News

Edgewise Therapeutics has received $95 million in funding to advance the clinical development of its experimental therapy EDG-5506, designed to improve physical functioning in people with Becker muscular dystrophy ... Read more

December 17, 2020December 17, 2020

Genethon Cleared to Launch French Trial Testing GNT0004 Gene Therapy for DMD

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The French National Agency for Medicines and Health Products Safety has cleared Généthon to begin a clinical trial testing the safety and efficacy of GNT0004, a potential gene therapy ... Read more

December 15, 2020December 15, 2020

Lower-dose SRP-5051 Potentially More Effective Than Exondys 51, Phase 2 Trial Finds

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Sarepta Therapeutics’ investigational treatment SRP-5051 enhanced cell penetration, exon skipping, and dystrophin levels at a lower dose than its Exondys 51 (eteplirsen) therapy in a Phase 2 clinical trial ... Read more

December 10, 2020December 10, 2020

Translarna Approved in Russia for DMD Patients 2 and Older

News

Translarna (ataluren) has been approved in Russia for treating Duchenne muscular dystrophy (DMD) caused by nonsense mutations in patients ages 2 and older. “Translarna was the first therapeutic ever approved ... Read more

December 8, 2020December 8, 2020

Lilly, Precision to Develop Gene Editing Therapies for DMD, Other Disorders

News

A new collaboration between Eli Lilly and Precision BioSciences aims to develop gene editing therapies for genetic disorders, initially focusing on Duchenne muscular dystrophy (DMD) and two other undisclosed ... Read more