Potential treatment for Niemann-Pick type C, a rare neurodegenerative disease
Por um escritor misterioso
Last updated 21 janeiro 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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