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 nonsense mutation Duchenne muscular dystrophy patients and we are excited to expand approval of Translarna into Russia,” Stuart…

A new collaboration between Eli Lilly and Precision BioSciences aims to develop gene editing therapies for genetic disorders, initially focusing on Duchenne muscular dystrophy (DMD) and two other undisclosed targets, the two companies announced. Precision licensed its ARCUS genome editing platform to Lilly and will lead…

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 federally approved COVID-19 vaccine. The MDA made its request in a letter to members of the…

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…