Author: Andi Thornton
Guidance released by Clinical Panel regarding accessing Nusinersen (Spinraza) out of area
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
Rebels win Spinraza battle – everybody can now experience the force
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”.
Rebels win Spinraza battle – everybody can now experience the force
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”.
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 neuron 2 (SMN2) gene. Weight ≥ 8.5 kg and ≤ 21 kg at the time…
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.