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 – ...
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Santhera launches DMD therapy in the UK following NICE nodNICE’s positive final guidance for Agamree follows confirmation that no appeals were submitted against its positive December ...
The FDA needs to strengthen guardrails to ensure its accelerated approval pathway is used appropriately, the HHS Office of ...
The results suggest that prolonging ambulation may not adversely impact cardiac function in adulthood for patients with ...
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 ...
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 ...
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