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.