Author: Marni Smyth
Sibs: Camp in the Cloud for siblings and their families
A SMA community member has sent us the following information, they thought it would be useful for the SMA community.
Zolgensma Q&A
We have received further information from NHSE on Zolgensma. We will continue to update you all as we receive information. Please follow link below for the Q&A. Zolgensma Q and As
MHRA Approve Licence for Risdiplam
Today the MHRA have approved the licence for Risdiplam.
NHSE announce Zolgensma treatment centres
We are please to share communication from NHSE about the progress regarding access to Zolgensma. The selection of the four centres which will be administering the treatment has been completed.
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
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!