You can hear about the wonderful impact of gene therapy, Zolgensma, for babies with Spinal Muscular Atrophy (SMA) Type 1 which has recently been used for the first time in Ireland – on RTE’s Hospital Live here. (It starts 8.47 minutes in.) The programme features parents, David Ryan and Liz McMahon, as well as Dr Declan […]
86 per cent of parents want to know if their newborn has a rare condition, even if it’s not yet treatable
Rare Diseases Ireland has published a new research study which reveals that 86 per cent of Irish parents want to know if their newborn has a rare condition, even if it is not yet treatable. 65 per cent of parents are in favour of screening newborns for as many rare conditions as possible. The study, […]
Parent Project Muscular Dystrophy (PPMD) Conference 23-26 June 2022
Parent Project Muscular Dystrophy’s (PPMD) 2022 Annual Conference takes place from 23-26 June virtually, as well as in-person in Arizona. The virtual conference experience will feature: Presentations and discussion panels featuring the latest research, clinical trials, approved therapies, care initiatives, quality of life issues, and more. In-depth presentations and discussion panels on gene therapy and […]
