Results 131 to 140 of about 3,855 (201)

An Indirect Treatment Comparison of Avalglucosidase Alfa versus Cipaglucosidase Alfa Plus Miglustat in Patients with Late-Onset Pompe Disease. [PDF]

Adv Ther
Roberts ME, Proskorovsky I, Guyot P, Shukla P, Thibault N, Hamed A, Pulikottil-Jacob R, O'Callaghan L, Pollissard L.   +8 more
europepmc   +1 more source

Elevated Cerebrospinal Fluid Total Tau in Niemann-Pick Disease Type C1: Correlation With Clinical Severity and Response to Therapeutic Interventions. [PDF]

J Inherit Metab Dis
Cawley NX, Zhou R, Farhat NM, Iben J, Alexander DM, Luke RA, Padilla CJ, Mohamed HO, Albert OK, Robbins KP, Rahhal S, Do AD, Berry-Kravis E, Cologna SM, Liu F, Porter FD.   +15 more
europepmc   +1 more source

New drugs: Miglustat

Australian Prescriber, 2010
openaire   +1 more source

Early Diagnosis of Nieman-Pick Disease Type C and Rapid Response of Gelastic Cataplexy to Treatment With N-Acetyl-L-Leucine: A Case Report. [PDF]

Am J Case Rep
Rojnueangnit K, Puttamanee S, Intarakhao S, Khusiwilai K.   +3 more
europepmc   +1 more source

Enzyme replacement therapy compared with best supportive care for the treatment of Pompe Disease: a systematic review and network meta-analysis.

Health Technol Assess
Corbett M, Umemneku-Chikere C, Nevitt S, Deng NJ, Walton M, Fulbright H, Tan CY, Lachmann R, Churchill R, Hodgson R.   +9 more
europepmc   +1 more source

Biomarker Validation in NPC1: Foundations for Clinical Trials and Regulatory Alignment. [PDF]

J Inherit Metab Dis
Casazza K, Cologna SM, Berry-Kravis E, Jarnes J, Porter FD.   +4 more
europepmc   +1 more source

Niemann-Pick C-like Endolysosomal Dysfunction in DHDDS Patient Cells, a Congenital Disorder of Glycosylation, Can Be Treated with Miglustat. [PDF]

Int J Mol Sci
Best HL, Cook SR, Waller-Evans H, Lloyd-Evans E.   +3 more
europepmc   +1 more source

Enzyme replacement therapies in adults with Pompe disease: from trials to real-world data. [PDF]

Curr Opin Neurol
van der Beek NAME, Potters LH, Schoser B.   +2 more
europepmc   +1 more source

Targeting Glucosylceramide Synthase: Innovative Drug Repurposing Strategies for Lysosomal Diseases. [PDF]

Int J Mol Sci
Canini G, Mazzinelli E, Nocca G, Lattanzi W, Arcovito A.   +4 more
europepmc   +1 more source

<i>N</i>-Alkyl Derivatives of Deoxynojirimycin (DNJ) as Antiviral Agents: Overview and Update. [PDF]

Molecules
Checconi P, Iacopetta D, Catalano A, Ceramella J, Cavalluzzi MM, Mariconda A, Marsico S, Aquaro S, Longo P, Sinicropi MS, Lentini G.   +10 more
europepmc   +1 more source