PTC Therapeutics recently revealed the 5 winners of its inaugural global awards program, the STRIVE Awards (Strategies to Realize Innovation, Vision…
Articles by Margarida Azevedo, MSc
The FSH Society, a nonprofit, patient-driven organization supporting research and education for facioscapulohumeral muscular dystrophy (FSHD), one of the most prevalent…
BioMarin Pharmaceutical, Inc., a company that develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions, has officially launched kNOWyourDuchenne, a…
Duchenne muscular dystrophy (DMD) is the most frequently inherited human myopathy and, as the name implies, is characterized by wasting…
Arizona-based Critical Path Institute (C-Path), a leading non-profit organization committed to accelerate and reduce developmental costs of medical products, recently announced it has formed…
Eleven year-old mountaineer, Tyler Armstrong, first fell in love with conquering the outdoors when he was 7 years old. Today, he…
In a recent study published in the Journal of Pediatric Ophthalmology and Strabism, a team of researchers…
Santhera Pharmaceuticals, a Swiss pharmaceutical company specialized in developing and commercializing breakthrough treatments for orphan mitochondrial and neuromuscular diseases, announced that it…
Researchers have been exploring the use of stem cell therapeutics for the treatment of severe conditions such as type 1 diabetes, Alzheimer’s…
A scientist who helped develop a pioneering treatment recently expressed her frustration after the NHS refused funding of Translarna for the…