Results 101 to 110 of about 2,627 (188)

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  

Miglustat in Neuronopathic Lysosomal Storage Disorders: Biological Rationale, Clinical Evidence, and Limits of Repurposing. [PDF]

Int J Mol Sci
Lipiński P, Węgrzyn G.
europepmc   +1 more source

Aspiration and silent aspiration in Niemann-Pick disease type C1: longitudinal findings from the NIH natural history study. [PDF]

Orphanet J Rare Dis
Solomon BI, Munoz AM, Borruso A, Sinaii N, Farhat N, Alexander D, Labor DA, Do AD, Porter FD.   +8 more
europepmc   +1 more source

Defining the therapeutic corridor of stability in enzyme replacement therapy for Pompe disease: a position statement. [PDF]

Orphanet J Rare Dis
Schoser B.
europepmc   +1 more source

Correction: Miglustat as a Treatment for Adults with Tangier Disease Neuropathy: The MUSTANG N‑of‑1 Trial with 21 months Clinical Observation. [PDF]

Neurol Ther
Cook A, Stuart B, Ochoa-Ferraro A, Condon N, Lawrence M, Webley F, Wallom KL, Forbes C, Veeranna V, Wanninayake S, Oliver T, Davies N, Dawson C, Platt F, Geberhiwot T.   +14 more
europepmc   +1 more source

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

Assessing Surface Binding Events on Golgi Microsomes Isolated from Cancer Cells by Capillary Electrophoresis. [PDF]

Anal Chem
Davoine C, Cobraiville G, Pochet L, Fillet M.   +3 more
europepmc   +1 more source

A disease progression model comparing the long-term mobility and respiratory outcomes of adults with late-onset Pompe disease receiving cipaglucosidase alfa plus miglustat versus alglucosidase alfa. [PDF]

J Comp Eff Res
Dymond A, Green W, Crabtree M, MacCulloch A, Johnson N, Shohet S, Gielen V, Clarke S, Deegan P, Diaz-Manera J.   +9 more
europepmc   +1 more source

Comparing the efficacy of cipaglucosidase alfa plus miglustat with alglucosidase alfa for late-onset Pompe disease: an expanded network meta-analysis utilizing patient-level and aggregate data. [PDF]

J Comp Eff Res
Fu S, Hummel N, Shohet S, Johnson N, MacCulloch A, Castelli J, Kerr W, Fox B, Gielen V.   +8 more
europepmc   +1 more source

From Neonatal Cholestasis to Progressive Neurological Impairment: A Case of Niemann-Pick Disease Type C. [PDF]

Cureus
Carvalho de Sousa L, Gomes Pereira C, Magalhães C.   +2 more
europepmc   +1 more source