Long-Term 30-Month Results of N-Acetyl-L-Leucine for Niemann-Pick Disease Type C (P11-7.003)

Key Points

• The aim is to evaluate how N-acetyl-L-leucine affects individuals with Niemann-Pick disease type C over a long duration.
• Evaluated participants with Niemann-Pick disease type C over a 30-month period.
• Administered N-acetyl-L-leucine as an intervention.
• N-acetyl-L-leucine showed positive long-term effects in Niemann-Pick disease type C.
• Significant improvements in specific neurological functions were observed.