AveXis discloses data on AVXS-101 efficacy in treating early onset spinal muscular atrophy
AVXS-101 shows amazing effects in treating spinal muscular atrophy.
Category: Updates
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
The Rainbowfish clinical trial of risdiplam has started.
A new clinical trial opens in presymptomatic babies
The Rainbowfish clinical trial of risdiplam has started.
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.
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.
