News

August 20, 2020August 20, 2020

First Patients Enroll in Phase 1 Trial of EPM-01, Potential Becker MD Therapy

News

A Phase 1 clinical trial of EPM-01 ((+)-epicatechin), a potential oral treatment of Becker muscular dystrophy that targets cellular energy, has enrolled its first patients. This one-year study of Epirium Bio‘s small ... Read more

August 18, 2020August 18, 2020

Phase 3 Trial of Pamrevlumab Enrolling DMD Patients Unable to Walk

News

The LELANTOS Phase 3 clinical trial, which is evaluating FibroGen‘s pamrevlumab in people with Duchenne muscular dystrophy (DMD) who are no longer able to walk, has enrolled a first ... Read more

August 17, 2020August 17, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

News, syndicated

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. ... Read more

August 13, 2020August 13, 2020

FDA Approves Viltolarsen, Now Viltepso, for DMD Patients With Exon 53 Mutation

News

The U.S. Food and Drug Administration (FDA) has conditionally approved NS Pharma‘s Viltepso (viltolarsen) for people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Viltepso is given ... Read more

August 11, 2020August 11, 2020

Patient Feedback Being Sought on Puldysa for Possible NHS Funding

News

The National Institute for Health and Care Excellence (NICE) is urging patient organizations to gather feedback from those with Duchenne muscular dystrophy (DMD) who received Santhera Pharmaceuticals‘ investigational treatment ... Read more

August 10, 2020August 10, 2020

New Streaming Channel Showcases Rare Disease Films

News, syndicated

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the ... Read more

August 6, 2020August 6, 2020

MD Advocates Offer Advice for Students With Neuromuscular Disorders Amid COVID-19

News

The importance of communication and self-care for people with muscular dystrophy and other neuromuscular diseases who are pursuing their education during the COVID-19 pandemic was highlighted in a Facebook ... Read more

August 4, 2020August 4, 2020

FDA Requests More Data, Maintains Clinical Hold on IGNITE DMD Trial of SGT-001 Gene Therapy

News

The U.S. Food and Drug Administration (FDA) is maintaining the clinical hold on the Phase 1/2 trial testing Solid Biosciences’ gene therapy SGT-001 for people with Duchenne muscular dystrophy (DMD), the company ... Read more

July 30, 2020August 14, 2020

FDA Places SRP-9001, DMD Micro-dystrophin Gene Therapy, on Fast Track

News

An investigational gene therapy for Duchenne muscular dystrophy (DMD), called SRP-9001 micro-dystrophin, was given fast track designation by the U.S. Food and Drug Administration (FDA) its developer, Sarepta Therapeutics, announced in ... Read more

July 28, 2020July 28, 2020

Stem Cell Therapy VTA-110 Named FDA Orphan Drug for DMD Treatment

News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Vita Therapeutics‘ VTA-110, a candidate therapy for people with Duchenne muscular dystrophy (DMD). The designation grants ... Read more

Pin It on Pinterest