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 ...

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

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

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 ...

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

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

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

Leerink Partners analyst Joseph Schwartz has maintained their bullish stance on SLDB stock, giving a Buy rating yesterday.Stay Ahead of the ...

A trial team led by Latham’s Mike Morin and David Frazier and Finnegan’s William (Bill) Raich helped Sarepta Therapeutics ...

Of the 24 drugs, three approvals deviated from the others -- aducanumab; the synthetic hormone hydroxyprogesterone caproate ...

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