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1 . Academic Journal
Identification of a novel fusion Iduronidase with improved activity in the cardiovascular system
저자
by
Sarah Kim
;
Michael J. Przybilla
;
Chester B. Whitley
;
Li Ou
;
Mahmoud Al-Kofahi
, et al.
소스
Molecular Genetics and Metabolism Reports, Vol 33, Iss , Pp 100917- (2022)
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7 . Academic Journal
Chitotriosidase as a biomarker for gangliosidoses
저자
by
Sarah Kim
;
Chester B. Whitley
;
Jeanine R. Jarnes
.
소스
Molecular Genetics and Metabolism Reports, Vol 29, Iss , Pp 100803- (2021)
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5 . Academic Journal
Examination of a blood-brain barrier targeting β-galactosidase-monoclonal antibody fusion protein in a murine model of GM1-gangliosidosis
저자
by
Michael J. Przybilla
;
Christine Stewart
;
Timothy W. Carlson
;
Li Ou
;
Brenda L. Koniar
, et al.
소스
Molecular Genetics and Metabolism Reports, Vol 27, Iss , Pp 100748- (2021)
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7 . Academic Journal
Patients with Gaucher disease display systemic oxidative stress dependent on therapy status
저자
by
Reena V. Kartha
;
Marcia R. Terluk
;
Roland Brown
;
Abigail Travis
;
Usha R. Mishra
, et al.
소스
Molecular Genetics and Metabolism Reports, Vol 25, Iss , Pp 100667- (2020)
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7 .
Physiologically‐Based Pharmacokinetic Model Development, Validation, and Application for Prediction of Eliglustat
Drug–Drug
Interactions
저자
by
Siddhee A. Sahasrabudhe
;
Shen Cheng
;
Mahmoud Al‐Kofahi
;
Jeanine R. Jarnes
;
Neal J. Weinreb
, et al.
소스
Clinical Pharmacology & Therapeutics
. 112:1254-1263
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7 .
Long-term intravenous galsulfase in Maroteaux-Lamy syndrome further reduces urine glycosaminoglycans (GAG) after hematopoietic stem cell transplantation and improves cardiac function and endurance
저자
by
Alia Ahmed
;
Elizabeth Braunlin
;
Chester B. Whitley
;
Jeanine R. Jarnes
.
소스
Molecular Genetics and Metabolism
. 138:106993
Web of Science
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6 .
Resolving variants of unknown significance and pseudodeficiency alleles
저자
by
Nishitha R. Pillai
;
Jeanine R. Jarnes
;
Alia Ahmed
;
Todd Vanyo
;
Chester B. Whitley
.
소스
Molecular Genetics and Metabolism
. 138:107272
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7 .
Updated interim safety, biomarker, and efficacy data from Imagine-1: A phase 1/2 open-label, multicenter study to assess the safety, tolerability, and efficacy of a single dose, intra-cisterna magna (ICM) administration of PBGM01 in subjects with type I (early onset) and type IIA (late onset) infantile GM1 gangliosidosis (GM1)
저자
by
Jeanine R. Jarnes
;
Caroline A. Hastings
;
Can H. Ficicioglu
;
Debra-Lynn Day-Salvatore
;
Roberto Giugliani
, et al.
소스
Molecular Genetics and Metabolism
. 138:107169
Web of Science
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7 .
Prospective longitudinal study of neurological disease trajectory in children living with late-infantile or juvenile onset of GM1 or GM2 gangliosidosis (PRONTO study)
저자
by
Roberto Giugliani
;
Benedicte Heron
;
Jeanine R. Jarnes
;
Marc C. Patterson
;
Maurizio Scarpa
, et al.
소스
Molecular Genetics and Metabolism
. 135:S47-S48
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7 .
WORLDSymposiumTM 2022⁎
저자
by
Chester B. Whitley
;
Lalitha Belur
;
Brian Bigger
;
Elizabeth Braunlin
;
Philip J. Brooks
, et al.
소스
Molecular Genetics and Metabolism
. 135:S1
Web of Science
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