Niemann-Pick B - RS - 5th infusion.

Current Clinical Applications of In Vivo Gene Therapy with AAVs: Molecular Therapy

Niemann-Pick C1 expression is not regulated by the amount of cholesterol flowing through cells in the mouse - ScienceDirect

IJMS, Free Full-Text

New Treatment of Disease that Swells Organs Gives New Hope to 4-year-old Girl - International Niemann-Pick Disease Alliance (INPDA)

Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis, Orphanet Journal of Rare Diseases

Sphingomyelin-induced inhibition of the plasma membrane calcium ATPase causes neurodegeneration in type A Niemann–Pick disease

PDF) Defects in sarcolemma repair and skeletal muscle function after injury in a mouse model of Niemann-Pick type A/B disease

Niemann-Pick B - RS - 5th infusion.

Lysosomal storage diseases: Diagnostic confirmation and management of presymptomatic individuals

TAVI in Patient Suffering from Niemann–Pick Disease (Acid Sphingomyelinase Deficiency) with Concomitant Situs Inversus and Dextrocardia

Pediatric hepatocellular carcinoma associated with Niemann–Pick disease type C: Case report and literature review - Hwang - 2023 - JIMD Reports - Wiley Online Library

Lysosomal storage diseases: Diagnostic confirmation and management of presymptomatic individuals

PDF) Intestinal Absorption of -Tocotrienol Is Mediated by Niemann-Pick C1-Like 1: In Situ Rat Intestinal Perfusion Studies

Niemann-Pick disease type C

Investigating the Mechanism of Cyclodextrins in the Treatment of Niemann‐ Pick Disease Type C Using Crosslinked 2‐Hydroxypropyl‐β‐cyclodextrin - Carradori - 2020 - Small - Wiley Online Library