Results 81 to 90 of about 14,662 (177)

Optimization of systemic AAV9 gene therapy in Niemann-Pick disease, type C1 mice. [PDF]

Life Sci Alliance
Mylvara AV, Gibson AL, Gu T, Davidson CD, Incao AA, Melnyk K, Gembic SR, Pierre-Jacques D, Cologna SM, Venditti CP, Porter FD, Pavan WJ.   +11 more
europepmc   +1 more source

Niemann-Pick disease type A

, 2018
Frank Gaillard, Daniel Bell, Craig Hacking   +2 more
openaire   +1 more source

Dysregulation of Extracellular Vesicle Concentration, MicroRNAs, and Surface Proteins in Patients With Niemann-Pick Disease Type C. [PDF]

J Extracell Biol
Hawthorne SCB, Sandau US, McFarland TJ, Berry-Kravis E, Saugstad JA.   +4 more
europepmc   +1 more source

THE FDA HAS APPROVED A DRUG AGAINST NIEMANN–PICK DISEASE, TYPE C

Педиатрическая фармакология
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Article Editorial
doaj  

Arimoclomol in infants with Niemann-Pick disease type C: Results from the phase 2/3 open-label pediatric substudy. [PDF]

Mol Genet Metab Rep
Mengel E, Arash-Kaps L, Grunewald S, Weller Grønborg S, Berger N, Shammas H, Dali CÍ.   +6 more
europepmc   +1 more source

Characterization of liver disease in a cohort of individuals with Niemann-Pick Disease, Type C1. [PDF]

Mol Genet Metab
Agrawal N, Bianconi S, Jaeger R, Farhat NY, Alexander DM, Sinaii N, Hadigan C, Berry-Kravis E, Porter FD.   +8 more
europepmc   +1 more source

Zuclopenthixol inhibits residual activity of acid sphingomyelinase in Niemann pick disease type B: a case report with in vitro validation. [PDF]

Orphanet J Rare Dis
Schiller M, Steiner KM, Bonnet U, Gulbins E, Scherbaum N.   +4 more
europepmc   +1 more source

Extracellular vesicles in Niemann pick disease type C: current knowledge and future opportunities. [PDF]

Front Cell Neurosci
Hawthorne SCB, Sandau US, Saugstad JA.
europepmc   +1 more source

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

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