N-Acetyl-leucine in progressive CACNA1A ataxia: A case series

In all children treated with NAL, NAL resulted in rapid improvement of ataxia, (gait, balance, fine motor and speech – mean SARA improvement at first follow-up: 3.25 points). Improvement was sustained up to 3 years (mean long-term SARA improvement: 5.13 points). SCAFI and CGI-I showed similar improvement. NAL was well-tolerated, without adverse reactions.

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