News

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

News

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

November 10, 2020November 10, 2020

AMO-02 for Congenital DM1, Entering Pivotal Trial, Gets Rare Pediatric Disease Status

News

The U.S. Food and Drug Administration (FDA) has given rare pediatric disease status to AMO Pharma‘s investigational therapy AMO-02 (tideglusib), now entering clinical testing in children and adolescents with congenital myotonic dystrophy ... Read more

November 5, 2020November 5, 2020

Ultragenyx and Solid Biosciences Partner on DMD Gene Therapy

News

Ultragenyx Pharmaceutical and Solid Biosciences announced a strategic collaboration to develop and commercialize new gene therapies for Duchenne muscular dystrophy (DMD). The collaboration will combine Solid’s gene therapy construct for ... Read more

November 4, 2020November 6, 2020

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

News, syndicated

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit ... Read more

November 3, 2020November 3, 2020

PPMD Seeking Adults with Duchenne or Becker MD to Join Advisory Committee

News

Parent Project Muscular Dystrophy (PPMD) is seeking adults with Duchenne or Becker muscular dystrophy to serve on the 2021 PPMD Adult Advisory Committee (PAAC). The committee is meant to ... Read more