Three new animal-model tests of a potential gene therapy for oculopharyngeal muscular dystrophy (OPMD), known as BB-301, are…
Articles by José Lopes, PhD
The U.S. Food and Drug Administration (FDA) has granted fast track designation to suvodirsen as a treatment for…
BBP-418, an oral treatment candidate for patients with limb-girdle muscular dystrophy type 2I (LGMD2I), is being developed by…
The European Medicines Agency (EMA) has validated a marketing authorization application for Puldysa (idebenone) for treating respiratory dysfunction in Duchenne…
Limb-girdle muscular dystrophy (LGMD) may be caused by previously unidentified TTN gene variants that are also associated with…
A protein called epidermal growth factor (EGF) can help preserve muscle strength and increase muscle regeneration in a mouse model…
Audentes Therapeutics is expanding its pipeline of potential molecular therapies, expecting to address 80% of patients with…
Using a genetic engineering tool to restore dystrophin while raising levels of utrophin, a similar protein, leads to better improvements…
All three children with limb girdle muscular dystrophy type 2E (LGMD2E) treated with the investigational gene therapy MYO-101 in a…
The investigational treatment edasalonexent was well-tolerated, led to no safety issues, and blocked a crucial muscle disease protein in…