Defeat Duchenne Canada Marks 30 Years of Progress with Major Research Funding Announcement
Defeat Duchenne Canada is proud to celebrate its 30th anniversary, marking three decades of relentless progress in the fight ...
Struggling With Muscle Weakness? It May Be A Sign Of Muscular Dystrophy
Muscular Dystrophy (MD) is a genetic condition causing progressive muscle weakness and complications in vital organs. Early ...
WebMD5d
Duchenne Muscular Dystrophy
What Is Duchenne Muscular Dystrophy? Muscular dystrophies are a group of diseases that make muscles weaker and less flexible over time. Duchenne muscular dystrophy (DMD) is the most common type.
pharmaphorum4d
Santhera preps UK Duchenne drug launch after NICE says yes
Santhera Pharmaceuticals will launch its new drug for Duchenne muscular dystrophy in the UK within the next few weeks, after ...
Family of Birmingham girl with one in 50 million condition say 'we want to do everything that we can'
Matilda Baptiste is 16 from Hall Green needs help to buy vital equipment to help her live independently with Duchenne ...
PMLiVE4d
Santhera’s Duchenne muscular dystrophy drug Agamree accepted by SMC
Santhera Pharmaceuticals’ Agamree (vamorolone) has been accepted by the Scottish Medicines Consortium (SMC) to treat Duchenne ...
The American Journal of Managed Care7d
Later Loss of Ambulation Linked With Better Heart Health in DMD
The results suggest that prolonging ambulation may not adversely impact cardiac function in adulthood for patients with ...
Solid Biosciences Outpaces Competitors In Duchenne Gene Therapy Development (CORRECTED)
Solid Biosciences advances gene therapies for muscular and cardiac diseases, gaining FDA clearance for SGT-212 and targeting ...
Santhera Receives Positive NICE Final Guidance for AGAMREE® (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy
Santhera has begun launch preparations for AGAMREE® in UK with first sales expected in this quarterPratteln, Switzerland, January 16, 2025 – ...
The American Journal of Managed Care6d
Quantifying the Altruism Value for a Rare Pediatric Disease: Duchenne Muscular Dystrophy
Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 ...
Solid Biosciences: Strategic Advancements and Promising Pipeline Justify Buy Rating
Leerink Partners analyst Joseph Schwartz has maintained their bullish stance on SLDB stock, giving a Buy rating yesterday.Stay Ahead of the ...
WebMD5d
Muscular Dystrophy
Muscular dystrophy is caused by defects in certain genes, with type determined by the abnormal gene. In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne ...