The SIDEROS Phase 3 clinical trial, investigating if oral Puldysa (idebenone) can be added to a stable glucocorticoid regimen to more effectively…
Articles by Inês Martins, PhD
The U.S. Food and Drug Administration (FDA) is requesting more information on the process used to manufacture the gene therapy …
People with Duchenne and Becker muscular dystrophy should not forego their routine health assessments during the COVID-19 pandemic, but are encouraged…
CureDuchenne Ventures has given $1 million in seed funding to support the development of…
Bamboo Therapeutics has been fully acquired by Pfizer, considerably expanding that company’s holdings in gene therapy. With the acquisition, Pfizer…
A new study from the University of Liverpool, has identified high amounts of a neutrophil-derived protein in the muscle of…
Ryan Benton, the first patient to be approved in the United States for allogenic adult stem cell transplantation as a Duchenne…
Nicotinamide riboside (NR), a compound that has been shown effective in boosting metabolism, can also induce muscular regeneration and enhance…
Genea Biocells has developed a human stem cell-based cellular model for a type of muscular dystrophy, known as facioscapulohumeral…
Researchers have discovered the molecular mechanisms responsible for the heart dysfunctions that mark myotonic dystrophy, and reported they appear to be linked to…