I posted this in Chat and someone suggested this board too. I'm asking people to please sign and share this petition.
www.change.org/p/nicola-sturgeon-ensure-access-to-duchenne-drug-translarna-in-scotland
There is a rare disease called Duchenne Muscular Dystrophy (DMD). It mainly affects boys. They are generally diagnosed around age 5, in a wheelchair by age 10 and then in their teens vital organs including the lungs and heart degenerate. They usually have a lifespan of somewhere in their 20s.
Some boys have been on a trial for a drug called Translarna, which slows the muscle breakdown and is the first and only drug which treats the underlying cause of DMD. The boys are still walking without the need for a wheelchair, including one who is 16!
The trial has been successful and NICE has approved it for use in England, Wales and NI. The Scottish Medical Consortium declined it meaning boys in Scotland cannot get this drug on the NHS. It costs £200k/year for each boy. The families of the Scottish boys are heartbroken, it is their only hope of keeping their children mobile and eligible for future treatments currently in development. Please help the families to petition the Scottish Govt. to make this drug available. Thank you mumsnetters.