News

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…

Seven pharmaceutical companies are banding together to sponsor Project Hercules — an initiative that addresses evidence generation for Duchenne muscular dystrophy (DMD) treatments. The project, led by the non-profit organization Duchenne UK, aims to simplify the way evidence is generated for health technology assessments by authorities such as Great Britain’s National…

Researchers have found that Duchenne muscular dystrophy patients usually have simultaneous neurological disorders or abnormalities, including epilepsy. Particularly, those who had epilepsy also had the neurodevelopmental condition attention deficit hyperactivity disorder (ADHD), or the neuropsychiatric conditions obsessive-compulsive disorder, anxiety or sleep disorders. Since the research, published in the European…

Clinical data from a Phase 3 trial revealed that Kyndrisa (drisapersen) could not significantly improve the walking ability of boys with Duchenne muscular dystrophy (DMD) due to mutations amenable with exon 51 skipping. Still, additional analysis of the DEMAND III trial (NCT01254019) showed this therapy may still…

Researchers have succeeded in growing working muscle tissue from a skin sample — a feat that until recently was viewed as science fiction. Although the lab-grown muscle was not as strong as those in the body, the advance opens up for new approaches to tissue regeneration in patients with muscular dystrophy…

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…

Apple Tree Partners has invested $40 million in Stoke Therapeutics to come up with gene-expression treatments for muscular dystrophy and other genetic disorders. Stoke aims to develop antisense oligonucleotides to promote targeted augmentation of nuclear gene output (TANGO). The goal is to reverse the features of rare diseases…

Expansion Therapeutics has raised $55.3 million in Series A financing to advance its portfolio of drugs targeting several genetic disorders, including myotonic dystrophy type 1 (DM1), the leading cause of adult onset muscular dystrophy. Toxic levels of RNA are thought to…

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…

Researchers tracked the way adult skeletal muscle stem cells (MuSCs) multiply in response to aging or injury. The study has implications for treatments in diseases of muscle tissues, such as muscular dystrophies. The study, “Muscle Stem Cells Exhibit Distinct Clonal Dynamics in Response to Tissue Repair and Homeostatic…