Potential treatment for Niemann-Pick type C, a rare neurodegenerative disease
Por um escritor misterioso
Last updated 15 abril 2025

Researchers in Japan have improved a potential treatment for a rare genetic disease, decreasing its negative toxic effects by threading it onto a dumb-bell-shaped chain and holding it in place until it reaches its target.

Estimating the prevalence of Niemann-Pick disease type C (NPC) in

Niemann-Pick type C: contemporary diagnosis and treatment of a

Patient Files: Niemann-Pick type C - PharmaTimes Magazine April 2018

Recommendations for the detection and diagnosis of Niemann-Pick

IJMS, Free Full-Text

Clinical patient groups with an increased risk of Niemann-Pick

Treatment outcomes following continuous miglustat therapy in

Ocular motor biomarkers in Niemann-Pick disease type C: A

Diagnostic workup and management of patients with suspected

Correlation of age of onset and clinical severity in Niemann–Pick
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