Campaign for Access to Treatments for Spinal Muscular Atrophy

NICE emailed us that they are not able to update us yet.

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.

So I guess our silence is fairly obvious. We have heard nothing from NICE, not a single word.

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!

We are all waiting for NICE's decision on Spinraza which is planned for tomorrow.

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 are working with other charities like TreatSMA on a policy project to improve access to medicines for rare conditions.

Recruitment in the first clinical trial of branaplam is complete.