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1 .
Colaboradores
저자
by
Abzug, Mark J.
;
Adams, David R.
;
Adelson, Stewart L.
;
Aiken, John J.
;
Akay, Begum
, et al.
소스
In
Nelson Tratado de Pediatria
Edition: Twentieth Edition. 2018:xiv-xliii
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7 .
Contributors
저자
by
Abzug, Mark J.
;
Adams, David R.
;
Adelson, Stewart L.
;
Aiken, John J.
;
Akay, Begum
, et al.
소스
In
Nelson Textbook of Pediatrics
Edition: Twentieth Edition. 2016:vi-xxxv
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7 . Academic Journal
Neuropsychological implications of Cobalamin C (CblC) disease in Hispanic children detected through newborn screening.
저자
by
Whitaker, Ashley M.
;
Thomas, Nina Hattiangadi
;
Krivitzky, Lauren S.
;
Ficicioglu, Can H.
.
소스
Applied Neuropsychology: Child; Apr-Jun2018, Vol. 7 Issue 2, p143-149, 7p
Web of Science
Scopus
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6 . Academic Journal
RGX-111 gene therapy for the treatment of severe mucopolysaccharidosis type I (MPS I): Interim analysis of data from the first in human study.
저자
by
Wang, Raymond Y.
;
Ficicioglu, Can H.
;
Giugliani, Roberto
;
Burke, Jenna
;
Fiscella, Michele
, et al.
소스
Molecular Genetics & Metabolism
. Feb2023, Vol. 138 Issue 2, pN.PAG-N.PAG. 1p.
Full Text (ScienceDirect)
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7 . Academic Journal
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
Jarnes, Jeanine R.
;
Hastings, Caroline A.
;
Ficicioglu, Can H.
;
Day-Salvatore, Debra-Lynn
;
Giugliani, Roberto
, et al.
소스
Molecular Genetics & Metabolism
. Feb2023, Vol. 138 Issue 2, pN.PAG-N.PAG. 1p.
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7 . Academic Journal
RGX-121 gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II): Interim analysis of data from the first in human study.
저자
by
Harmatz, Paul
;
Ficicioglu, Can H.
;
Giugliani, Roberto
;
Rajan, Deepa
;
Hagood, Joseph
, et al.
소스
Molecular Genetics & Metabolism
. Feb2023, Vol. 138 Issue 2, pN.PAG-N.PAG. 1p.
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7 . Academic Journal
Retinal Structure in Cobalamin C Disease: Mechanistic and Therapeutic Implications.
저자
by
Aleman, Tomas S.
;
Brodie, Frank
;
Garvin, Christopher
;
Gewaily, Dina Y.
;
Ficicioglu, Can H.
, et al.
소스
Ophthalmic Genetics
. Dec2015, Vol. 36 Issue 4, p339-348. 10p.
Web of Science
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7 . Academic Journal
Liver Pathology in Infantile Mitochondrial DNA Depletion Syndrome.
저자
by
HAZARD, FLORETTE K.
;
FICICIOGLU, CAN H.
;
GANESH, JAYA
;
RUCHELLI, EDUARDO D.
.
소스
Pediatric & Developmental Pathology; Nov/Dec2013, Vol. 16 Issue 6, p415-424, 10p
Web of Science
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6 . Academic Journal
Cobalamin C Deficiency Shows a Rapidly Progressing Maculopathy With Severe Photoreceptor and Ganglion Cell Loss
저자
by
Bonafede, Lucas
;
Ficicioglu, Can H.
;
Serrano, Leona
;
Han, Grace
;
Morgan, Jessica I. W.
, et al.
소스
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE
; DEC 2015, 56 13, p7875-p7887, 13p.
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7 . Academic Journal
Cobalamin C Deficiency Shows a Rapidly Progressing Maculopathy with Severe Ganglion Cell Loss
저자
by
Aleman, Tomas S.
;
Bonafede, Lucas
;
Mills, Monte D.
;
Forbes, Brian J.
;
Binenbaum, Gil
, et al.
소스
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE
; JUN 2015, 56 7, 3p.
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