Duchenne muscular dystrophy is a serious condition that causes progressive muscle weakness. It affects 2,500 boys and men in ...

Duchenne muscular dystrophy (DMD) is the most common type ... It was the first targeted treatment for this type of mutation and has been shown to help increase the production of dystrophin.

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 ...

Muscular dystrophy is caused by defects in certain genes, with type determined by the abnormal gene. In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne ...

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

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

Wales and Northern Ireland for the treatment of Duchenne muscular dystrophy (DMD) in patients 4 years of age and older. This follows confirmation that no appeals were received against the Final ...