A document has been released by the Clinical Panel regarding accessing Nusinersen (Spinraza) out of area. You can read the full document below. Please note this information is for England only. Nusinersen referrals guidance FINAL
So it’s officially Star Wars day, those of you who haven’t woken up fully or have no idea what we are talking about, May the 4th is referred to by many geeks as Star Wars day due to the phrase made famous by the series, “may the force be with you”.
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)