The experimental therapy vamorolone can improve motor function significantly in boys with Duchenne muscular dystrophy (DMD), without stunting ...

Real-world and natural history data could be used to supplement, or perhaps replace, the use ...

Sarepta Therapeutics has completed a meeting with the Office of Tissues and Advanced Therapies (OTAT) ...

An imbalance in cells producing proteins called aldehyde dehydrogenases (ALDHs) — favoring fat formation in ...

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is ...

An application requesting the approval of casimersen (SRP-4045) to treat Duchenne muscular dystrophy (DMD) amenable to exon 45 ...

NS Pharma‘s Viltepso (viltolarsen) is now commercially available in the U.S. for people with Duchenne ...

A Phase 1 clinical trial of EPM-01 ((+)-epicatechin), a potential oral treatment of Becker muscular dystrophy that ...

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to ...

The U.S. Food and Drug Administration (FDA) has conditionally approved NS Pharma‘s Viltepso (viltolarsen) for ...

The National Institute for Health and Care Excellence (NICE) is urging patient organizations to gather ...

The U.S. Food and Drug Administration (FDA) is maintaining the clinical hold on the Phase 1/2 trial ...

An investigational gene therapy for Duchenne muscular dystrophy (DMD), called SRP-9001 micro-dystrophin, was given fast track designation ...

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Vita Therapeutics‘ ...

Benitec Biopharma has begun the first study to support the start of clinical trials of ...

Two magnetic resonance measures — MRI and magnetic resonance spectroscopy (MRS) — of leg muscles are ...

NS Pharma has launched a support hub, called NS Support, for people in the U.S. with ...

Hansa Biopharma has granted an exclusive worldwide license to Sarepta Therapeutics to develop and promote ...

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease ...

Oral Puldysa (idebenone), Santhera Pharmaceuticals‘ investigational treatment for breathing problems caused by Duchenne muscular dystrophy ...

Sarepta Therapeutics has submitted a full application to the U.S. Food and Drug Administration (FDA) ...

The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, ...

A Phase 1 clinical trial of BBP-418, a potential first oral therapy for limb-girdle muscular dystrophy ...

A partnership between Muscular Dystrophy Canada (MDC) and Technology for Living (TFL) will seek to ...

At the one year mark, Sarepta Therapeutics’ SRP-9001 micro-dystrophin gene therapy continues to show positive ...

The European Commission has granted orphan drug designation to viltolarsen, a treatment for people with ...

A single administration of the gene therapy SRP-9003 at a low dose continues to lead ...

Santhera Pharmaceuticals announced the signing of two agreements with Rutgers University related to the development of ...

Parent Project Muscular Dystrophy (PPMD) and Duchenne UK have joined forces to offer up to ...

NS Pharma’s lead candidate viltolarsen safely and effectively increases dystrophin levels and promotes improvements in boys with Duchenne ...

A Phase 2 trial of ATL1102 met its primary goal of demonstrating favorable safety and tolerability ...

The SIDEROS Phase 3 clinical trial, investigating if oral Puldysa (idebenone) can be added to a stable glucocorticoid ...

Pfizer’s experimental gene therapy PF-06939926 promotes sustained production of a shorter but functional version of ...

One-year treatment with CAP-1002, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy (DMD) significantly improves respiratory, cardiac ...

The U.S. Food and Drug Administration (FDA) is requesting more information on the process used ...

I’ve never considered myself to be insecure about my disability. My use of a mobility ...

CureDuchenne Ventures has given $1 million in seed funding to support the development of Myosana Therapeutics‘ ...

Both PTC Therapeutics and Sarepta Therapeutics expect to continue providing an uninterrupted supply of their Duchenne ...

Author’s note: The COVID-19 crisis has already lasted several months. The reality is that we ...

The Muscular Dystrophy Association (MDA) has formed a Medical Advisory Team to provide guidance related ...

Netflix’s new documentary “Crip Camp” transports viewers to the peak era of peace-preaching hippies and ...

Traci and Tony Rico of Cardiff-by-the-Sea, California, juggle more than their share of family health ...

None of us have lived through something like what we are going through today. Some ...

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Epirium Bio’s ...

Capricor Therapeutics will host a conference call and webcast with a cardiac expert to discuss ...

A CureDuchenne Ventures equity investment will support Dyne Therapeutics in developing precision treatments aimed at repairing muscles in ...

In the days when I still attended school, I was deemed an extrovert by my ...

Invitae and the Muscular Dystrophy Association (MDA) are partnering to expand access to free genetic ...

I’ve always sought silver linings, including in this season of social distancing. Many people now ...

I feel strange not writing about the pandemic crisis in this column, but we’re already ...

Final results of a Phase 1 clinical trial support the safety of losmapimod and suggest ...

NS Pharma launched an expanded access program (EAP) in the U.S. to allow certain Duchenne muscular dystrophy ...