SMA-I-002

Spinal Muscular Atrophy (SMA)

Asia 6 months female South Korea Total Cost: 1772

Patient Condition

Three months after birth, the child developed hypotonia, weak crying, and reduced limb activity. Type SMA-I was diagnosed 5 months later, with a homozygous deletion of exon7 in the SMN1 gene, and a copy number of SMN2 was 3. He relied on nasal feeding at the time of his visit and was unable to turn over. His HINE-2 score was 8/26, and he needed intermittent oxygen inhalation.

Treatment Timeline

PhaseDetailsDate
Assessment and Risdiplam treatment initiation
Risdiplam combined with Nusinersen
Rehabilitation training and follow-up

Treatment Outcome

beforeHINE-2 8 points, relying on nasal feeding, unable to turn over and raise his head, intermittent oxygen inhalation
afterHINE-2 26 points (full score), sit and stand independently, swallow normally, get out of nasal feeding
highlightThe HINE-2 score reached full marks, eating independently from nasal feeding, achieving a milestone recovery