Author: Andi Thornton
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.
Biogen Nurture study reveals new and positive data
Nurture Study reveals new and positive data showing benefits of treating pre-symptomatic infants with Spinraza at the annual congress of the World Muscle Society.
Biogen Nurture study reveals new and positive data
Nurture Study reveals new and positive data showing benefits of treating pre-symptomatic infants with Spinraza at the annual congress of the World Muscle Society.
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.
TreatSMA met with the Roche UK team
On Monday 24 September, TreatSMA held a meeting with Roche UK to discuss our advocacy work and the company’s plans related to risdiplam.