Zolgensma NHSE Announcement
An agreement between NHSE and Novartis Gene Therapies has been reached to enables families in England to have access to gene therapy for Spinal Muscular Atrophy.
TreatSMA take part in the Royal Holloway’s Annual Rare Disease Day (online) event
Today TreatSMA took part in the Royal Holloway’s Annual Rare Disease Day (online) event speaking to students about Uni Life and Rare Disease. It was great to spread awareness of SMA and rare diseases and also answer some great questions!
Roche receives positive CHMP opinion for Evrysdi (Risdiplam)
Roche receives positive CHMP opinion for Evrysdi, the first and only at home spinal muscular atrophy (SMA) treatment with proven efficacy in adults, children and infants two months and older.
Government Launch the new UK Rare Diseases Framework
A new UK Rare Diseases Framework has been published demonstrating the commitment of the UK Government to improving the lives of people who have rare conditions.
Pathfinders Neuromuscular Alliance are hosting a Natural Nutrition Event
Pathfinders Neuromuscular Alliance are hosting a Natural Nutrition event on Monday 15th March at 1pm.
Shielding Guidance extended until 31st March 2021.
Shielding guidance for those who are clinically extremely vulnerable has been extended now until the 31st March 2021, instead of the previous date given 21st February 2021.
Update from NHS England regarding risdiplam EAMS
This morning we have received an update from NHS England making it easier to understand who is and isn’t eligible for treatment under the EAMS programme.
TreatSMA are representing the community at the NICE appraisal of Zolgensma (onasemnogene abeparvovec-xioi).
TreatSMA are today representing the community at the NICE appraisal of the first Gene Therapy for SMA, Zolgensma (onasemnogene abeparvovec-xioi).