The clinicians and SMA community react to the draft nusinersen guidance
We are trying to find a way out of the MAA criteria, please stay tuned.
NICE is refusing to amend the entry criteria for Spinraza treatment. We are fighting on.
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.
Closer to a new treatment: NICE has begun the appraisal process of SMA gene therapy Zolgensma.
NICE releases the final draft of the Managed Access Agreement. We will challenge some of the treatment criteria it proposes.
The gene therapy drug Zolgensma (AVXS-101) receives FDA approval to treat spinal muscular atrophy in children below 24 months.
AveXis presented data that shows astonishing efficacy of AVXS-101 in SMA type 2 children