Muscular Dystrophy News

January 14, 2021January 14, 2021

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

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 first ... Read more

January 12, 2021January 13, 2021

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

Muscular Dystrophy News

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 motor ... Read more

January 7, 2021January 7, 2021

Pivotal Trial Begins for AMO-02 in Steinert Disease

COVID-19 News, Muscular Dystrophy News

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 adolescents ... Read more

January 5, 2021January 5, 2021

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

Muscular Dystrophy 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 (BMD) ... Read more

December 17, 2020December 17, 2020

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

Muscular Dystrophy News

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 for ... Read more

December 15, 2020December 16, 2020

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

Muscular Dystrophy News

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 in ... Read more

December 10, 2020December 10, 2020

Translarna Approved in Russia for DMD Patients 2 and Older

Muscular Dystrophy 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 for ... Read more

December 8, 2020December 8, 2020

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

Muscular Dystrophy News

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

December 4, 2020January 5, 2021

MDA Wants Neuromuscular Disease Patients to Have Early Access to COVID-19 Vaccine

COVID-19 News, Muscular Dystrophy News

The Muscular Dystrophy Association (MDA) is encouraging the U.S. Centers for Disease Control and Prevention (CDC) to recommend that people living with neuromuscular diseases (NMDs) have early access to any ... Read more

December 3, 2020December 3, 2020

ReveraGen Receives $3.3M NIH Grant to Advance Vamorolone in DMD

Muscular Dystrophy News

ReveraGen BioPharma has been awarded a $3.3 million grant from the National Institute of Neurological Disorders and Stroke (NINDS) at the National Institutes of Health (NIH) to support further development ... Read more

December 2, 2020January 3, 2021

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

Muscular Dystrophy News

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) ... Read more

December 1, 2020December 2, 2020

Sarepta Talks Progress, Plans for New Trials of DMD Gene Therapy SRP-9001

COVID-19 News, Muscular Dystrophy News

Sarepta Therapeutics is about to launch a Phase 1 trial of SRP-9001, its investigational gene therapy for Duchenne muscular dystrophy (DMD), and is planning other clinical studies for the near ... Read more

November 26, 2020December 28, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

COVID-19 News, Muscular Dystrophy News

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a ... Read more

November 24, 2020November 24, 2020

DM1 Patients Asked to Join Natural History Study Aiming for Better Clinical Trials

Muscular Dystrophy News

The Myotonic Dystrophy Clinical Research Network (DMCRN) is asking adults with myotonic dystrophy type 1 (DM1) to participate in a large natural history study aimed at improving clinical trials by ... Read more