Campaign for Access to Treatments for Spinal Muscular Atrophy

We are asking the SMA community, friends and family to wear in public on the 7th May a plain t-shirt or top with a personal message to NHS England and NICE telling them we will not wait any longer.

NICE has not yet been able to recommend Spinraza, and an urgent Committee meeting has been called.

An Expanded Access Programme of Spinraza starts in Russia today.

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.