Author: TreatSMA
Biogen statement re. EAP in England, Wales and Northern Ireland
Biogen has provided an update for the SMA Community on the Expanded Access Programme to nusinersen.
SMA to become a terminal condition again in England and Wales
Tonight at midnight the UK Expanded Access Programme to Spinraza will end for children diagnosed with SMA henceforth, and with it SMA will become a terminal disease again.
Julian Sturdy MP asks the Health Secretary about Spinraza
Julian Sturdy MP has asked the Health Secretary about access to Spinraza treatment.
TreatSMA Families make final plea inside and outside NICE
Vigil held outside NICE whilst others inside make last desperate fight for treatment.
TreatSMA vigil on the decisive day
Today the National Institute of Health and Care Excellence is to decide whether spinal muscular atrophy will be treated on the NHS.
Avexis files for regulatory approval of gene therapy
Avexis has submitted applications for regulatory approval of AVXS-101, the groundbreaking gene therapy product developed to treat spinal muscular atrophy.
NICE started appraisal procedure for SMA gene therapy
The National Institute of Health and Care Excellence has begun procedures leading to appraisal of AVXS-101, the first gene therapy to treat spinal muscular atrophy.
Roche announces positive interim results of risdiplam trials
Today Roche announced interim clinical data from the dose-finding parts of the FIREFISH and SUNFISH clinical trials investigating risdiplam as a treatment for spinal muscular atrophy.