On this Rare Diseases Day we are thrilled to announce that Translarna has been approved in Ireland! We are so grateful for all your support and prayers in making this happen. We hope and pray that this drug will make a great difference to the young boys who need it. Our joy has been dampened a little by the news that those suffering with SMA have been refused treatment of the drug Spinraza which has been shown to be effective. Please consider signing their petitions to help them: https://www.change.org/p/approve-access-to-miracle-drug-spinraza-in-ireland https://www.change.org/p/simon-harris-oireachtas-ie-approve-new-miracle-drug-spinraza-for-sma-patients-in-ireland-immediately