Author Archives: Inês Martins PhD

The SIDEROS Phase 3 clinical trial, investigating if oral Puldysa (idebenone) can be added to a stable glucocorticoid regimen to more effectively slow lung function decline in boys and men with Duchenne muscular dystrophy (DMD), has completed enrollment, Santhera Pharmaceuticals announced. A planned analysis demonstrated the study, while not enrolled up to the…

The U.S. Food and Drug Administration (FDA) is requesting more information on the process used to manufacture the gene therapy SGT-001, as it reviews the clinical hold placed on the Phase 1/2 trial of this Duchenne muscular dystrophy (DMD) therapy candidate, Solid Biosciences reported. IGNITE DMD (NCT03368742) will remain…

People with Duchenne and Becker muscular dystrophy should not forego their routine health assessments during the COVID-19 pandemic, but are encouraged to transition their care to telemedicine and to medical tests performed at home to minimize risk. Those are among the consensus recommendations provided by neuromuscular specialists to help these patients…

CureDuchenne Ventures has given $1 million in seed funding to support the development of Myosana Therapeutics‘ potential non-viral gene therapy for people with Duchenne muscular dystrophy (DMD). The money is expected to advance a platform that delivers the full DMD gene to the muscles…

Bamboo Therapeutics has been fully acquired by Pfizer, considerably expanding that company’s holdings in gene therapy. With the acquisition, Pfizer gains several potential therapeutic agents for rare diseases, including one for Duchenne muscular dystrophy (DMD), soon to enter clinical testing; and one Phase 1/2 clinical stage agent, a potential best-in-class recombinant Adeno-Associated Virus (rAAV)…

A new study from the University of Liverpool, has identified high amounts of a neutrophil-derived protein in the muscle of a Duchenne muscular dystrophy (DMD) mouse model. The finding shows that the protein hinders stem cells from repairing damaged muscle and it reveals a potential drug target for DMD therapies. The…

Ryan Benton, the first patient to be approved in the United States for allogenic adult stem cell transplantation as a Duchenne muscular dystrophy (DMD) treatment, turned 30 this year — largely exceeding his original life expectancy. DMD is a genetic disease that affects 1 out of every 3,600 male infants, leading to…

Nicotinamide riboside (NR), a compound that has been shown effective in boosting metabolism, can also induce muscular regeneration and enhance life span in mice, according to the study “NAD+ repletion improves mitochondrial and stem cell function and enhances life span in mice,” published in…

Genea Biocells has developed a human stem cell-based cellular model for a type of muscular dystrophy, known as facioscapulohumeral muscular dystrophy (FSHD), that may be a suitable tool for high-throughput screening and the development of therapeutic agents for the disease. The model is described in the study “A…

Researchers have discovered the molecular mechanisms responsible for the heart dysfunctions that mark myotonic dystrophy, and reported they appear to be linked to a defective processing of the cardiac sodium channel (SCN5A) RNA. Their study, published in Nature Communications, is titled “Splicing misregulation of SCN5A contributes to cardiac-conduction delay and…