NICE responds to us
NICE emailed us that they are not able to update us yet.
France decides to offer Spinraza to all
Today the French Ministry of Health authorised wide reimbursement of Spinraza. Now every SMA patient in France will be able to receive life-saving treatment.
NICE silence on Spinraza approval. TreatSMA demands action from NHS England
So I guess our silence is fairly obvious. We have heard nothing from NICE, not a single word.
AveXis discloses data on AVXS-101 efficacy in treating early onset spinal muscular atrophy
Avexis, the company behind gene replacement therapy AVXS-101, disclosed preliminary data from their phase-3 clinical trial of intravenous AVXS-101 (Zolgensma). The effects are simply breathtaking!
Will NICE recommend Spinraza tomorrow?
We are all waiting for NICE's decision on Spinraza which is planned for tomorrow.
A new clinical trial opens in presymptomatic babies
As communicated by Roche, the first site to run the Rainbowfish clinical trial had been activated and is ready to accept presymptomatic babies diagnosed with spinal muscular atrophy.
Genetic Alliance UK announces the “Resetting The Model” project
Genetic Alliance UK are working with other charities like TreatSMA on a policy project to improve access to medicines for rare conditions.
Recruitment completed in branaplam trial
Recruitment in the first clinical trial of branaplam is complete.