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 ...
pharmaphorum3d
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 ...
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 ...
PMLiVE3d
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 ...
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 – ...
Parent Project Muscular Dystrophy Hosts 2025 Duchenne Healthcare Professionals Summit in San Diego, California
Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), will host its sixth in-person Duchenne Healthcare Professionals ...
The American Journal of Managed Care6d
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 ...
Brandon Wechsler's inspiring legacy celebrated at Pinnacle basketball game
Wheeling around the gym, always smiling, always chatting it up with players, Brandon Wechsler brought a charm and positivity ...
The American Journal of Managed Care5d
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 ...