A new clinical trial is starting for gene therapy for patients aged 3 months - 20 years old who are: Symptomatic SMA diagnosis based on gene mutation analysis with bi-allelic survival motor neuron 1 (SMN1) mutations (deletion or point mutations)…
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
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.