News

December 4, 2020December 4, 2020

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

News, syndicated

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 ... Read more

December 3, 2020December 3, 2020

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

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 ... Read more

December 2, 2020December 2, 2020

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

News, syndicated

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 ... Read more

December 1, 2020December 4, 2020

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

News

Note: This story was updated Dec. 4, 2020, to note that Teji Singh’s title is executive medical director of Sarepta, rather than the chief medical officer. Sarepta Therapeutics is ... Read more

November 26, 2020November 26, 2020

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

News, syndicated

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 ... Read more

November 24, 2020November 24, 2020

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

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 ... Read more

November 19, 2020November 19, 2020

Phase 1 Trial of Oral EDG-5506 for Becker MD Enrolling at Texas Site

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Edgewise Therapeutics announced the opening of a Phase 1 clinical trial to assess the safety and tolerability of EDG-5506, its investigational oral treatment for Becker muscular dystrophy (BMD). The ... Read more

November 18, 2020November 18, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

News, syndicated

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about ... Read more

November 17, 2020November 17, 2020

Q&A: Meet the Muscular Dystrophy Association’s New CEO and President

News

The Muscular Dystrophy Association (MDA) announced last week it had selected former MDA vice chairman of the board Donald Wood, PhD, as the new president and CEO of the ... Read more

November 12, 2020November 12, 2020

MDA Launches Tool to Make Clinical Data More Accessible to Researchers

News

The Muscular Dystrophy Association (MDA) and DNAnexus have introduced a digital platform to improve patient care and accelerate treatment discovery for muscular dystrophy and other neuromuscular disorders. The neuroMuscular ObserVational ... Read more