Case report of a 71-year-old female patient successfully treated with nusinersen

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.


Elderly patient with 5q spinal muscular atrophy type 4 markedly improved by Nusinersen. Morizumi T, Ueno A, Takasone K, Ozawa K1, Yoshinaga T, Nakamura K, Sekijima Y. J Neurol Sci. 2020 (Mai). 415:116901.