Duchenne Muscular Dystrophy

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No parent should be told their child has a life-limiting condition over a video call
Update on reaching out to decision makersPetition Update 💙 We've now reached 743 signatures – thank you to every single person who has signed and shared. Every signature …
29 June 2026
TIME IS MUSCLE - Keep Our Boys Stronger for Longer
Update on Givinostat for Irish Duchenne boysWhere Things Stand NowBig update: the NCPE has completed its review of Givinostat, and negotiations are now underway — but every d…
16 March 2026
Please make Translarna available in Ireland to help boys with Duchenne Muscular Dystrophy
Victory!On this Rare Diseases Day we are thrilled to announce that Translarna has been approved in Ireland! We are so grateful for all you…
28 February 2019

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