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Living with CMT has limited areas of my life. Some of the everyday things that people without my…
News
The National Institute for Health and Care Excellence’s (NICE) have published draft guidance ‒…
Blog
On Sunday 15 October Angela, with her husband Brian, will officially start Bidwells Cambridge Town…
News
On Friday, 22 September, PTC Therapeutics, the company that manufactures Translarna, issued a…
News
Translarna currently has a conditional marketing approval (authorisation) from the EMA, given in…
Blog
I decided to get involved with fundraising for Muscular Dystrophy UK (MDUK) as my dad, Edward,…
Blog
“Today has definitely been one for the memory box.”
Louisa, whose son, Archie, lives with…
Blog
Running for Andrew
Graham recently decided to get back into running after a long break and last…
News
Our new research grants cover a variety of different muscle-wasting conditions, such as ADSSL1…
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NICE recommendations
Although not NICE's final guidance on this medicine, the draft guidance…