In a letter to the editor published in May 2020, a Japanese team reports their experience of treatment with nusinersen of the oldest patient treated to date. This 71-year-old female patient has SMA type 4. She has a deletion in the SMN1 gene and 4 copies of the SMN2 gene. Her first motor problems appeared at the age of 56. Nusinersen treatment for 3 months improved the fatigability of this patient, who was able to walk again without the aid of a cane.
While this is only a special case, this finding in favor of the effectiveness of nusinersen in SMA type IV is encouraging.
