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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 of vamorolone…

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) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

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 about to launch a Phase 1 trial of SRP-9001, its investigational gene therapy for Duchenne muscular dystrophy (DMD),…

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 survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

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 better understanding the full spectrum of the disorder. “Prior drug studies have been limited by a lack…

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 study (NCT04585464) is now enrolling up to 152 healthy volunteers and adults with BMD, ages 19 to 55, at…

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 how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…

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 organization, taking over from Lynn O’Connor Vos. Wood most recently was the vice president of institutional…

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 Research (MOVR) Visualization and Reporting Platform (VRP) provides easy-to-use tools for in-depth analysis of large neuromuscular patient…

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 type 1 (CDM1), also known as Steinert disease. “This Rare Pediatric Disease designation highlights the significant…