Results 121 to 130 of about 990 (176)

Inclusion body myositis - pathomechanism and lessons from genetics. [PDF]

Open Med (Wars), 2015
Murnyák B, Bodoki L, Vincze M, Griger Z, Csonka T, Szepesi R, Kurucz A, Dankó K, Hortobágyi T.   +8 more
europepmc   +1 more source

CDG Therapies: From Bench to Bedside. [PDF]

Int J Mol Sci, 2018
Brasil S, Pascoal C, Francisco R, Marques-da-Silva D, Andreotti G, Videira PA, Morava E, Jaeken J, Dos Reis Ferreira V.   +8 more
europepmc   +1 more source

457. Successful Use of Lipoplex for Delivery of the Small Molecules ManNAc and Sialic Acid To Rescue Hyposialylation in a Mouse Model of Hereditary Inclusion Body Myopathy (HIBM) [PDF]

Molecular Therapy, 2011
openaire   +1 more source

586. A Single Patient, Multiple Intravenous (IV) Infusion, Clinical Trial of GNE-Lipoplex for Therapy of Hereditary Inclusion Body Myopathy (HIBM)

Molecular Therapy, 2010
openaire   +1 more source

New Developments in the Genetics of Inclusion Body Myositis. [PDF]

Curr Rheumatol Rep, 2018
Britson KA, Yang SY, Lloyd TE.
europepmc   +1 more source

HNRNPA1 de novo Variant Associated with Early Childhood Onset, Rapidly Progressive Generalized Myopathy. [PDF]

J Neuromuscul Dis
Roos A, Häusler M, Kollipara L, Topf A, Preusse C, Stucka R, Nolte K, Strom T, Berutti R, Jiang X, Koll R, Lochmüller H, Schacht SM, Zahedi RP, Weis J, Senderek J.   +15 more
europepmc   +1 more source

578. Manufacturing and Stability Testing of a GNE-Lipoplex for a Clinical Trial for the Treatment of Hereditary Inclusion Body Myopathy Type (HIBM)

Molecular Therapy, 2010
openaire   +1 more source

Μοριακή διερεύνηση μιας οικογένειας με αυτοσωμική επικρατούσα κληρονομική μυοσίτιδα με έγκλειστα (HIBM)

, 2010
openaire   +1 more source

When a negative (charge) is not a positive: sialylation and its role in cancer mechanics and progression. [PDF]

Front Oncol
Habeeb IF, Alao TE, Delgado D, Buffone A.   +3 more
europepmc   +1 more source