Scotland’s NHS can now offer givinostat to ambulant children with Duchenne muscular dystrophy, marking a step forward in care ...

The Scottish Medical Consortium has approved the use of givinostat to treat children aged six and older who are still able to walk.

Cost regulators for the NHS in England and Wales have now issued final guidance recommending the use of PTC Therapeutics' Translarna for patients with Duchenne muscular dystrophy. Cost regulators for ...

NICE has given PTC Therapeutics extra time to hash out a cost-effective price plan with NHS England before it will allow a final yes for funding through the country’s state-run healthcare system. Just ...

Pratteln, Switzerland, January 14, 2025 – Santhera Pharmaceuticals (SIX: SANN) announces that the Scottish Medicines Consortium (SMC) has published recommendations that AGAMREE® (vamorolone) is ...

After months of delays, and despite a prominent campaign, NICE has announced it will not recommend funding on the NHS for breakthrough drug Translarna which could keep boys with life-limiting Duchenne ...

From reproductive rights to climate change to Big Tech, The Independent is on the ground when the story is developing. Whether it's investigating the financials of Elon Musk's pro-Trump PAC or ...

A new treatment to protect boys from a muscle-wasting disease that condemns them to life in a wheelchair – and an early death – is now available on the NHS in England. Studies suggest the ...

Archie Hill, who has Duchenne, met David Cameron last year to appeal for his support for the campaign for Translarna A drug for treating children aged five and over with Duchenne muscular dystrophy ...

For the first time ever, children will be able to access a drug tackling an underlying cause of life-limiting, Duchenne muscular dystrophy through NHS England. Translarna could keep children with the ...

Finley Hesketh's family were initially told that he will not be given a new drug which could delay his deteriorating health An eight-year-old boy with a rare muscle-wasting disease is "making huge ...

The watchdog which considers the costs of medicines for the NHS has recommended against funding a new treatment for Duchenne muscular dystrophy (DMD). The National Institute for Health and Care ...