A Phase 2 clinical trial of ATL1102, Antisense Therapeutics’ investigational therapy for Duchenne muscular dystrophy (DMD), recently received authorization to proceed from the Royal Children’s Hospital (RCH), Melbourne Human Research Ethics Committee. ATL1102 inhibits the protein CD49d, which is required for the normal function of the VLA-4 (very late…
Santhera Pharmaceuticals recently launched an expanded access program (EAP) to allow certain Duchenne muscular dystrophy (DMD) patients in the U.S. to gain access Raxone (idebenone). Raxone is not approved to treat Duchenne MD by the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA). The BreatheDMD…
The U.S. Food and Drug Administration (FDA) recently granted the gene therapy BB-301 orphan drug status for the treatment of oculopharyngeal muscular dystrophy (OPMD), according to Benitec Biopharma. OPMD is caused by a mutation of the poly(A)-binding protein nuclear 1 (PABPN1) gene. The resulting faulty protein can form…
Santhera Pharmaceuticals has started a campaign to help Duchenne muscular dystrophy patients and their families learn more about the breathing challenges the condition can pose. The company’s Take a Breath DMD campaign is focusing on the importance of providing patients with proper respiratory care. It began in the United States but will…
Through Dec. 31, Parent Project Muscular Dystrophy (PPMD) will match all donations to expand its ongoing Gene Therapy Initiative in an effort to raise $400,000 to fund research projects seeking to advance gene therapy as a treatment for Duchenne muscular dystrophy (DMD). Gene therapy has been increasingly studied…
Clay Matthews of the Green Bay Packers took to the field in Week 13 of the NFL season wearing a cause close to his heart on his feet, as he joined the football league’s “My Cause My Cleats” campaign to raise funds for Duchenne muscular dystrophy (DMD). “My…
The first healthy subject was dosed in a Phase 1b clinical trial evaluating the safety, tolerability and drug properties of ELX-02 as a candidate for the treatment of multiple conditions, including Duchenne muscular dystrophy (DMD). The multiple ascending dose (MAD) study (NCT03309605) is being led by Sevion…
Duchenne UK has teamed up with five pharmaceutical companies to start Project HERCULES, a collaboration aimed at persuading the national health insurance system to cover new Duchenne muscular dystrophy therapies. Ensuring that patients obtain access to innovative therapies is a two-part challenge. The first is obtaining regulatory approval of the drugs.
The U.S. Food and Drug Administration (FDA) has cleared an investigational new drug application for a micro-dystrophin gene therapy program by Sarepta Therapeutics and Nationwide Children’s Hospital, in clinical development for Duchenne muscular dystrophy (DMD). A Phase 1/2a clinical trial is now enrolling participants and should begin dosing…
The Hope for Gus Foundation and its signature Everest to End Duchenne fundraising trek to Mount Everest Base Camp have raised $100,000 to fund new gene-editing research that could lead to new treatments for Duchenne muscular dystrophy (DMD). The nine-member U.S. Everest to End Duchenne team…
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