Results 121 to 130 of about 21,027 (214)

Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study

, 2007
Miglustat for treatment of Niemann-Pick C disease: a randomised controlled ...
MC Patterson (14464527), D Vecchio (13425981), JE Wraith (14464524), Larry Abel (13056207), H Prady (13425984)   +4 more
core  

A whole-brain structural connectivity signature in adult Niemann-Pick disease type C. [PDF]

Brain Commun
Reilly TC, Di Biase MA, Mansour S, Seguin C, Cropley VL, Velakoulis D, Zalesky A, Pantelis C, Walterfang M.   +8 more
europepmc   +1 more source

Niemann-Pick Disease With Bilateral Adrenal Mass. [PDF]

JCEM Case Rep, 2023
Long Mohd Noor Affendi SNAB, Tong CV, Nordin NDB.   +2 more
europepmc   +1 more source

Miglustat in Niemann-Pick type C (NPC) disease: Results of 12-months-treatment

, 2007
Miglustat in Niemann-Pick type C (NPC) disease: Results of 12-months ...
D Vecchio (13425981), Marc Patterson (5636096), Larry Abel (13056207), H Prady (13425984)   +3 more
core  

Niemann–Pick Disease

, 2014
G.M. Pastores, H.A. Lau
  +4 more sources

Retention of lysosomal acid sphingomyelinase protects from Niemann-Pick Disease. [PDF]

Neurobiol Dis
Beard CA, Hermanson KN, Snider JM, Hara A, Dahl BK, Allopenna JJ, Marron MT, Newcomb B, Low BE, Wiles MV, Jenkins RW, Obeid LM, Hannun YA, Snider AJ.   +13 more
europepmc   +1 more source

Exploring the boundaries of Niemann-Pick disease type A/B: a report of a case and review of literature. [PDF]

Mol Cell Pediatr
El-Mezayen M, Tawfik AM, Hadhoud AM, Gerges VM, Afify MH.   +4 more
europepmc   +1 more source

Efficacy and safety of efavirenz in Niemann-Pick disease type C. [PDF]

Neurotherapeutics
Gascón-Bayarri J, Rico I, Sánchez-Castañeda C, Ledesma MD, Carnaval T, Bejr-Kasem H, Campdelacreu J, Ferrer A, Rodríguez-Bel L, Cos M, de Lama E, López de Munain A, Rouco I, Pérez-Sousa C, Cerdán M, Muelas N, Sevillano MD, Mir P, Villoria J, Videla S.   +19 more
europepmc   +1 more source

Evidence of Oxytosis/Ferroptosis in Niemann-Pick Disease Type C. [PDF]

Int J Mol Sci
Sanchez KL, Kim J, White JB, Tolan A, Rajagopal NP, Anderson DW, Shin AN, Shin SD, Currais A, Soriano-Castell D, Maher P, Soriano S.   +11 more
europepmc   +1 more source

Serum neurofilament light protein as a biomarker in Niemann-Pick disease, type C1. [PDF]

Genet Med Open
Cawley NX, Zhou R, Mylvara A, Padilla CJ, Alexander D, Farhat N, Alvarez C, Cougnoux A, Berry-Kravis E, Cologna SM, Liu F, Porter FD.   +11 more
europepmc   +1 more source