Potentially Protective Agent Shows Benefit in Niemann-Pick Type C
Patients with Niemann-Pick disease type C who received N-acetyl-L-leucine (NALL), an agent that potentially ameliorates lysosomal and metabolic dysfunction, showed reduced neurologic signs and symptoms after 3 months, a phase III crossover trial found. After 12 weeks of treatment, the mean change from baseline in scores on the Scale for the Assessment and Rating of […]