Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the…
Articles by Forest Ray PhD
A Phase 1 clinical trial of EPM-01 ((+)-epicatechin), a potential oral treatment of Becker muscular dystrophy that targets cellular energy, has…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Vita Therapeutics‘ VTA-110, a…
Benitec Biopharma has begun the first study to support the start of clinical trials of BB-301, a potential …
NS Pharma has launched a support hub, called NS Support, for people in the U.S. with Duchenne muscular dystrophy…
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure…
Sarepta Therapeutics has submitted a full application to the U.S. Food and Drug Administration (FDA) seeking approval of…
The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, has recommended a…
The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during…
People with neuromuscular disorders such as muscular dystrophy are not at a higher risk of acquiring COVID-19, but…