The SMA Community and clinicians react to draft NICE guidance
The clinicians and SMA community react to the draft nusinersen guidance
No update yet but please stay tuned
We are trying to find a way out of the MAA criteria, please stay tuned.
NICE refuses to reconsider Spinraza criteria
NICE is refusing to amend the entry criteria for Spinraza treatment. We are fighting on.
Spinraza treatment only for some?
We have analysed the NICE/NHS eligibility criteria for Spinraza and we view them as potentially depriving hundreds of children and adults of access to the only treatment. Read on.
NICE starts appraising Zolgensma to treat spinal muscular atrophy
Closer to a new treatment: NICE has begun the appraisal process of SMA gene therapy Zolgensma.
Draft Managed Access Agreement for Spinraza has been published
NICE releases the final draft of the Managed Access Agreement. We will challenge some of the treatment criteria it proposes.
FDA approves Zolgensma, the first gene replacement therapy for SMA
The gene therapy drug Zolgensma (AVXS-101) receives FDA approval to treat spinal muscular atrophy in children below 24 months.
Gene therapy works wonders in children with SMA type 2
AveXis presented data that shows astonishing efficacy of AVXS-101 in SMA type 2 children