Risdiplam is a pre-mRNA splicing modifier designed to treat 5q-SMA. We describe our experience prescrib- ing Risdiplam to adults with type-2 SMA with a wider range of ages and comorbidities than in clinical trials.

Risdiplam was prescribed for nine people aged 20 to 56-years; six males. Eight patients had homozygous exon-7 and 8 deletions; one had isolated homozygous exon-7 deletions. Seven patients had three copies of the SMN-2-gene, two had four copies. Seven patients had spinal surgery and two had severe scoliosis. They had between 0-5 comorbidities and 1-12 other drugs already prescribed and were all assessed for known interactions. They had blood tests (month 1, 3, and 6), ECG, and visual function assessment. Females of childbearing age were offered long-acting contraception and males were offered sperm banking.

Seven patients required non-invasive ventilation while two had dysphagia (one with gastrostomy). Baseline forced-Vital-Capacity (19%-74%), RUL (score 0-19), CHOP-ATTEND (score 19-46), and 9-hole-peg-test and dynamometer were measured as appropriate.

One experienced abdominal pain and increased weakness (Risdiplam was stopped). One patient had transaminitis and treatment was withheld and restarted later at a lower dose and is being gradually increased.

We found Risdiplam is generally well tolerated, especially for those with greater disability and spinal surgery.