News

PTC Therapeutics announced the start of its fourth annual STRIVE awards program for Duchenne muscular dystrophy (DMD). STRIVE — which stands for Strategies to Realize Innovation, Vision and Empowerment — supports programs run by nonprofit patient advocacy groups that aim to increase disease diagnosis, awareness and education, and train…

Treatment with edasalonexent (CAT-1004) showed sustained preservation of muscle function in a Phase 1/2 trial in boys with Duchenne muscular dystrophy (DMD), according to Catabasis Pharmaceuticals. The MoveDMD trial (NCT02439216) is investigating the safety and efficacy of edasalonexent in boys ages 4-7 at enrollment with any DMD-related mutation.

A new approach to CRISPR-Cas9 gene-editing technology, called myoediting, successfully restored dystrophin production and contraction force in heart muscle cells of Duchenne muscular dystrophy (DMD) patients. The new strategy, developed by U.S. and German researchers, targets sites located in “hot spots” of mutations along the dystrophin gene, allowing its…

The U.K.-based Duchenne Research Fund has granted £320,000 ($441,760) to researchers at Imperial College London to develop a bodysuit that may improve treatment decisions in Duchenne muscular dystrophy (DMD). The fund aims to help foster the development of new treatments for DMD by establishing collaborations between scientists, charities, and…

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…

CAP-1002, a cell therapy being developed by Capricor Therapeutics to treat Duchenne muscular dystrophy (DMD), has won a Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA). RMAT status is given to regenerative medicine therapies that show potential to treat serious conditions lacking available therapies.

European and Japanese researchers used an artificial chromosome to generate the dystrophin protein that’s missing in Duchenne muscular dystrophy. The chromosome delivered a two-gene package capable of producing the missing protein to a mouse model of the disease. The team also used it in a lab setting involving human cells.

The recently formed company Skyhawk Therapeutics announced it has obtained $8 million in seed funding to develop RNA-targeting small molecules for the treatment of over 50 diseases, including muscular dystrophy. The investment is led by “major family and biotech investors,” including Tim Disney, Alexandria Venture Investments, and the Duke of Bedford, among others,…

This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least 30 million Americans. The campaign kicks off Feb. 1 and culminates with Rare Disease Day on Feb. 28. Organized by…

An advisory committee of the European Medicines Agency (EMA) has issued another negative recommendation after re-examining a request to extend Raxone (idebenone) for use in treating Duchenne muscular dystrophy (DMD). The EMA’s Committee for Medicinal Products for Human Use (CHMP) issued its first negative opinion of the request in…