Results 141 to 150 of about 10,325 (203)

Apolipoprotein-mimetic nanodiscs reduce lipid accumulation and improve liver function in acid sphingomyelinase deficiency. [PDF]

Nanomedicine, 2023
Halseth TA, Correia AB, Schultz ML, Fawaz MV, Kuiper EQ, Kumaran P, Dorsey KH, Schuchman EH, Lieberman AP, Schwendeman A.   +9 more
europepmc   +1 more source

Correction to: Olipudase Alfa in Non-CNS Manifestations of Acid Sphingomyelinase Deficiency: A Profile of Its Use. [PDF]

Clin Drug Investig, 2023
Syed YY.
europepmc   +1 more source

A zebrafish model of combined saposin deficiency identifies acid sphingomyelinase as a potential therapeutic target

Disease Models & Mechanisms, 2023
Tejia Zhang, Ivy Alonzo, Chris Stubben, Yijie Geng, Chelsea Herdman, Nancy Chandler, Kim P. Doane, Brock R. Pluimer, Sunia A. Trauger, Randall T. Peterson   +9 more
doaj   +1 more source

Liver transplantation and portacaval shunt in genetic diseases [PDF]

, 1982
Starzl, TE
core  

Sphingolipids in cardiovascular diseases and metabolic disorders [PDF]

, 2015

core   +1 more source

Long-Term Safety and Clinical Outcomes With Olipudase Alfa Enzyme Replacement Therapy in Children and Adolescents With Acid Sphingomyelinase Deficiency. [PDF]

J Inherit Metab Dis
Scarpa M, Diaz GA, Giugliani R, Jones SA, Mengel E, Guffon N, Witters P, Ganesh J, Armstrong NM, Srivastava S, Kim Y.   +10 more
europepmc   +1 more source

Screening for lysosomal diseases in a selected pediatric population: the case of Gaucher disease and acid sphingomyelinase deficiency. [PDF]

Orphanet J Rare Dis, 2023
Di Rocco M, Vici CD, Burlina A, Venturelli F, Fiumara A, Fecarotta S, Donati MA, Spada M, Concolino D, Pession A.   +9 more
europepmc   +1 more source

Surgery for metabolic liver disease [PDF]

, 1986
Starzl, TE
core  

Real-life impacts of olipudase alfa: experiences of adults receiving enzyme replacement therapy for acid sphingomyelinase deficiency-results from an international survey study. [PDF]

Orphanet J Rare Dis
Morsy AS, Mbua S, Mathieson T, Hopkin J, Bolton S.   +4 more
europepmc   +1 more source

Olipudase Alfa in Non-CNS Manifestations of Acid Sphingomyelinase Deficiency: A Profile of Its Use. [PDF]

Clin Drug Investig, 2023
Syed YY.
europepmc   +1 more source