Author: Andi Thornton
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.
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.
Recruitment completed in branaplam trial
Recruitment to the branaplam trial has been completed.
Risdiplam trials: early results of Sunfish
Roche disclosed early data from part I of Sunfish trial – risdiplam
Risdiplam trials: exciting early results in Firefish
Roche disclosed early data from part I of Firefish trial – risdiplam
Irish families demand Spinraza NOW on World Rare Disease Day
Irish families will once again march on the Dáil this Thursday 28th February to demand the Health Service Executive approve Spinraza NOW.