EFFICACY AND SAFETY OF N-ACETYL-L-LEUCINE IN NIEMANN-PICK DISEASE TYPE

The IB1001-201 clinical trial with N-acetyl-L-leucine (IB1001) met its primary and secondary endpoints and demonstrated a statistically significant and clear clinically meaningful improvement in symptoms, functioning, and quality of life for pediatric and adult patients with NPC. IB1001 was well-tolerated and no drug-related serious adverse events were reported, demonstrating a favorable risk-benefit profile for the treatment of NPC.  Journal of Neurology.

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