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.