TreatSMA

New Zolgensma Clinical Trial

A new clinical trial is starting for gene therapy for patients who are:

Symptomatic SMA diagnosis based on gene mutation analysis with bi-allelic survival motor neuron 1 (SMN1) mutations (deletion or point mutations) and any copy of the survival motor neuron 2 (SMN2) gene.
Weight ≥ 8.5 kg and ≤ 21 kg at the time of Screening Visit 2
Naive to treatment or have discontinued an approved drug/therapy

You can read more on the trial here and please contact your neuromuscular specialist if you are interested.

Calling on all the community to ACT NOW to avoid losing treatments!
Currently, there are two approved treatments for SMA: risdiplam and nusinersen. These treatments were initially approved temporarily under Managed Access Agreements while the NHS and NICE gathered data to assess their cost-effectiveness. Now, as these agreements come to an end, both treatments face a crucial review.
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