Santhera has begun launch preparations for AGAMREE® in UK with first sales expected in this quarterPratteln, Switzerland, January 16, 2025 – ...
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 advances gene therapies for muscular and cardiac diseases, gaining FDA clearance for SGT-212 and targeting ...
IPS HEART has been awarded Orphan Drug Designation (ODD) by the FDA for GIVI-MPCs based on its unique ability to create new ...
The OIG report points to gaps in documentation and decision-making transparency, particularly in the case of Aduhelm, where FDA records indicated only 13 meetings with Biogen, but congressional ...
The Haslam family knew early on that their 10-year-old and 12-year-old were suffering from muscular dystrophy, but what they ...
Deramiocel is a cell therapy that has healing effects in muscle cells. If approved, deramiocel would be a once-quarterly ...
Sarepta Therapeutics’ Duchenne muscular dystrophy therapy Elevidys handily beat analysts’ expectations in the fourth quarter ...
Santhera Pharmaceuticals said that it received positive recommendation from the Scottish Medicines Consortium for use of AGAMREE ...
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported ...