Santhera Pharmaceuticals will launch its new drug for Duchenne muscular dystrophy in the UK within the next few weeks, after ...

The FDA established its accelerated approval program in 1992 to allow drugs to reach the market quickly for life-threatening ...

Santhera Pharmaceuticals’ Agamree (vamorolone) has been accepted by the Scottish Medicines Consortium (SMC) to treat Duchenne ...

Santhera has begun launch preparations for AGAMREE® in UK with first sales expected in this quarterPratteln, Switzerland, January 16, 2025 – ...

The FDA needs to strengthen guardrails to ensure its accelerated approval pathway is used appropriately, the HHS Office of ...

Solid Biosciences advances gene therapies for muscular and cardiac diseases, gaining FDA clearance for SGT-212 and targeting ...

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 ...

IPS HEART has been awarded Orphan Drug Designation (ODD) by the FDA for GIVI-MPCs based on its unique ability to create new ...

Sarepta Therapeutics’ Duchenne muscular dystrophy therapy Elevidys handily beat analysts’ expectations in the fourth quarter ...

The Haslam family knew early on that their 10-year-old and 12-year-old were suffering from muscular dystrophy, but what they ...

Santhera Pharmaceuticals said that it received positive recommendation from the Scottish Medicines Consortium for use of AGAMREE ...