Author: Marni Smyth
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)
Update on the Nusinersen (Spinraza) Eligibility Criteria Review
The nusinersen clinical eligibility criteria review is progressing as planned to the anticipated timeline.
Scottish Medicines Consortium Zolgensma Announcement
Scottish Medicines Consortium agreed access to the SMA Gene therapy Zolgensma
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.