Two of the three available seats for patient experts taken by TreatSMA trustees for the Risdiplam appraisal taking place on the 11th May 2021.
A new clinical trial is starting for gene therapy for patients who are: Symptomatic SMA diagnosis based on gene mutation analysis with bi-allelic survival motor neuron 1 (SMN1) mutations (deletion or point mutations) and any copy of the survival motor…
TreatSMA’s short film competition launches today, get your entries in by the 14th April to be in with a chance of winning a £50 Amazon voucher!
With the news regarding EMA Approval we are aware there may be some questions/concerns regarding EAMS. Please see below the following statement we have received from Roche.
New two-year data show Roche’s Evrysdi (risdiplam) continues to demonstrate improvement or maintenance of motor function in people aged 2-25 with Type 2 or Type 3 Spinal Muscular Atrophy (SMA)
Scottish Medicines Consortium agreed access to the SMA Gene therapy Zolgensma