Pharmacotherapy of Cerebellar Disorders, Including CACNA1A

We’re incredibly grateful to Dr. Michael Strupp for joining the CACNA1A Foundation to share the latest insights on ataxia treatments, especially his experience with NALL (N-Acetyl-L-Leucine), a modified amino acid. NALL is now FDA-approved under the name AQNEURSA for the treatment of Niemann-Pick Type C, another rare neurological disorder. In the video, Dr. Strupp notes that NALL’s safety and tolerability profile have been extensively studied in clinical trials, making it a promising candidate for the treatment of CACNA1A-related ataxia.
 In addition to the presentation recording, we would like to share highlights from the Q&A session that followed. Families asked excellent and thoughtful questions, and Dr. Strupp's answers were informative and full of hope.