Last Updated: 09/26/2026
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Found 3 publications
Long-term real-world safety and effectiveness of arimoclomol in individuals with NPC: Outcomes from the US early access program over a 4-year period.
Journal: Molecular genetics and metabolism
Published: April 13, 2026
Examining Epilepsy in Angelman Syndrome: Insights From Caregiver-Reported Data in the Linking Angelman and Dup15q Data for Expanded Research Database.
Journal: Pediatric neurology
Published: March 06, 2026
Case Report: The Association of Wilson Disease in a Patient With Ataxia and GLUT-1 Deficiency.
Journal: Frontiers in pediatrics
Published: July 30, 2021
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Last Updated: 09/26/2026