Case Progression:
At 8 years old, he began treatment with nusinersen when this treatment
became commercially available for SMA. He received a total of 8 doses.
He showed a modest improvement in his Hammersmith Functional Motor Scale
Extended (HFMSE) from the baseline of 6 to 9/66 after his first year of
therapy. At 9 years old, he was able to remain sitting with one hand
used for support and he could roll from his back to his side and flex
both hips while supine. The patient passed away at 10 years of age from
respiratory failure.