Santhera has begun launch preparations for AGAMREE® in UK with first sales expected in this quarter Pratteln, Switzerland, January 16, 2025 – (SIX: SANN) announces that the National Institute for ...

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 ...

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

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 ...

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

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

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

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

This year’s top stories on Duchenne muscular dystrophy (DMD) managed care included a novel imaging method for tracking disease progression, an FDA approval of the first nonsteroidal therapy ...

A groundbreaking study has shed light on the complex interactions between dystrophin, a protein critical to muscle stability, and its partner protein, dystrobrevin, offering new pathways for ...