Another TreatSMA Trustee to be on the Risdiplam Appraisal
Two of the three available seats for patient experts taken by TreatSMA trustees for the Risdiplam appraisal taking place on the 11th May 2021.
New Zolgensma Clinical Trial
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
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!
Further Updates Regarding EMA Approval and how it Affects EAMS
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.
Breaking News: European Medical Agency (EMA) Approve Evrysdi (Risdiplam)
European Medical Agency has recommended that Risdiplam can be used for treatment of SMA. The approval scope: 5q SMA over 2 months with clinical symptoms of SMA type 1, 2 or 3 OR with 1–4 copies of the SMN2 gene.
Roche Release New 2 Year Data for Evrysdi (Risdiplam)
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 Zolgensma Announcement
Scottish Medicines Consortium agreed access to the SMA Gene therapy Zolgensma