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Dosing Begins in Phase 3 Trial of Pfizer’s Gene Therapy for Duchenne
NewsThe first boy has been dosed in a Phase 3 trial testing Pfizer‘s gene therapy, PF-06939926, in treating Duchenne ... Read more -
I Make Sure My Support System Is Strong
columns, You, Me and MD - a Column by Leah LeilaniWhen I’m deep in thought, reflecting on my life, I shudder to think who and where I’d be without ... Read more -
DMD Boys Experience Biological, Functional Gains in SRP-9001 Study
NewsThe investigational gene therapy SRP-9001 safely leads to the production of a functional form of dystrophin in boys ... Read more -
NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum
News, syndicatedThe National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to ... Read more -
Why Freelancing Works for My Life With Duchenne
columns, Hawk's-Eye View - a Column by Hawken MillerSince my internship at The Washington Post, I’ve done freelance work at outlets ranging from The Forward, a Jewish-focused ... Read more -
Pivotal Trial Begins for AMO-02 in Steinert Disease
NewsAMO Pharma has announced the initiation of REACH-CDM, a pivotal Phase 2/3 clinical trial that will assess the efficacy ... Read more -
Top 10 Muscular Dystrophy Stories of 2020
NewsThroughout 2020, Muscular Dystrophy News Today brought you daily coverage of important discoveries, treatment advancements, clinical trial findings, ... Read more -
Edgewise Will Use $95M Funding to Advance EDG-5506 for BMD, DMD
NewsEdgewise Therapeutics has received $95 million in funding to advance the clinical development of its experimental therapy EDG-5506, designed ... Read more -
It’s Time to Spread Some Holiday Cheer
columns, Hawk's-Eye View - a Column by Hawken MillerChristmas is upon us, and for many, it might not even feel like the holiday season at all. We’ve ... Read more -
Genethon Cleared to Launch French Trial Testing GNT0004 Gene Therapy for DMD
NewsThe French National Agency for Medicines and Health Products Safety has cleared Généthon to begin a clinical trial testing ... Read more -
Lower-dose SRP-5051 Potentially More Effective Than Exondys 51, Phase 2 Trial Finds
NewsSarepta Therapeutics’ investigational treatment SRP-5051 enhanced cell penetration, exon skipping, and dystrophin levels at a lower dose than ... Read more -
Translarna Approved in Russia for DMD Patients 2 and Older
NewsTranslarna (ataluren) has been approved in Russia for treating Duchenne muscular dystrophy (DMD) caused by nonsense mutations in patients ages ... Read more
