José Lopes, PhD, managing science editor —

The investigational treatment Raxone (idebenone) could become available to qualifying Duchenne muscular dystrophy (DMD) patients in the U.S. with respiratory decline following the start of an expanded access program (EAP), Clinigen and Santhera Pharmaceuticals announced. BreatheDMD is an EAP authorized by the U.S. Food and Drug Administration…

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.

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…

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,…

Acceleron Pharma’s treatment for a muscular dystrophy affecting the face and other muscles increased the muscle mass of those with the disorder, according to a Phase 2 clinical trial. The results covered the first stage of the trial, which is assessing ACE-083’s3 ability to treat  facioscapulohumeral dystrophy, or FSHD. FSHD…

Researchers have developed a new “checkpoint” model that may be used to discover potential therapies for disorders such as Duchenne muscular dystrophy (DMD) and cystic fibrosis. The research, “Aminoglycoside-mediated promotion of translation readthrough occurs through a non-stochastic mechanism that competes with translation termination,” was published in the journal Human…

Successful results of Translarna (ataluren) and Emflaza (deflazacort) therapies for Duchenne muscular dystrophy (DMD) are highlighted in the 2017 second quarter report by PTC Therapeutics. Orally-administered Translarna is for Duchenne patients to reduce the effect of nonsense mutations in the DMD gene that lead to a lack of…

End-stage heart failure is increasingly becoming the main cause of death in patients with Duchenne muscular dystrophy (DMD). Because cardiomyopathy, an abnormal heart condition, is difficult to detect, a study examined current diagnostic methods, treatment options, and potential future therapies for heart failure among…

A proposed U.S. database of children with complex medical problems, including muscular dystrophy (MD), has raised concern at a respected public policy institute. Researchers at the Houston institute contend that the database could be used to discriminate against the children and their families, in particular to deny them health insurance. Dr.

One of a class of compounds that GTx is developing increased the survival of mice with Duchenne Muscular Dystrophy (DMD), and improved their muscle, heart and lung function, according to a pre-clinical trial study.   The therapies are known as selective androgen receptor modulators, or SARMs. The study, “Androgen Receptor Agonists…