Raymond Wang
机构: CHOC Children's Specialists / University of California-Irvine School of Medicine
主页: choc.org
每年引用次数
引用次数
引用: 3,804
H-指数: 32
I10-指数 : 66
出版物: 198
Free sialic acid storage disorder: Progress and promise
Marjan Huizing , Mary E Hackbarth , David R Adams , Melissa Wasserstein
Neuroscience letters 755 135896 -135896
20
2021
Amino acid metabolism
Raymond Y Wang , William R Wilcox , Stephen D Cederbaum
Emery Rimoin’s Essent. Med. Genet. 372 1 -42
13
2013
RGX-111 gene therapy for the treatment of severe mucopolysaccharidosis type I (MPS I): Interim analysis of data from the first in human study
Raymond Y Wang , Can H Ficicioglu , Roberto Giugliani , Jenna Burke
Molecular Genetics and Metabolism 138 ( 2) 107354 -107354
1
2023
Performance measures and patient/caregiver-reported assessments collected in a longitudinal, multi-center disease monitoring program (DMP) of patients with mucopolysaccharidosis type VII (MPS VII)
Betsy C Malkus , Jeanne M Cohen , Christina L Grant , Maurizio Scarpa
Molecular Genetics and Metabolism 141 ( 2) 107949 -107949
2024
Vestronidase alfa for the treatment of mucopolysaccharidosis type VII (MPS VII): Updated results from a novel, longitudinal, multi-center disease monitoring program (DMP)
Roberto Giugliani , Christina L Grant , Maurizio Scarpa , Angela Sun
Molecular Genetics and Metabolism 141 ( 2) 107851 -107851
2024
Vestronidase alfa for the treatment of mucopolysaccharidosis type VII (MPS VII): Updated results from a novel, longitudinal, multicenter Disease Monitoring Program (DMP)
Roberto Giugliani , Antonio Gonzalez-Meneses Lopez , Christina Grant , Maurizio Scarpa
Molecular Genetics and Metabolism 138 ( 2) 107117 -107117
2023
Cardiac responses in paediatric Pompe disease in the ADVANCE patient cohort
Barry J Byrne , Steven D Colan , Priya S Kishnani , Meredith C Foster
Cardiology in the Young 32 ( 3) 364 -373
8
2022
Collaborative research efforts drive therapeutic advancements for free sialic acid storage disorder (FSASD)
May Christine Malicdan , David R Adams , PJ Brooks , Konstantin Dobrenis
Molecular Genetics and Metabolism 141 ( 2) 107948 -107948
2024
disease type C; clinical experience in four cases [version 1; peer
William RH Evans , Elena-Raluca Nicoli , Raymond Y Wang , Nina Movsesyan
2017
disease type C; clinical experience in four cases
William RH Evans , Elena-Raluca Nicoli , Raymond Y Wang , Nina Movsesyan
2017
Problemas médicos únicos en pacientes adultos con mucopolisacaridosis
John Mitchell , Kenneth I Berger , Andrea Borgo , Elizabeth A Braunlin
Revista europea de medicina interna
The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII.
Raymond Y Wang , José Francisco da Silva Franco , Jaime López-Valdez , Esmeralda Martins
Molecular Genetics and Metabolism 129 ( 3) 219 -227
4
2020
Long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis type VII
Raymond Y Wang , JOSE FRANCO , Jaime López-Valdez , Esmeralda Martins
Molecular Genetics and Metabolism
2020
Corrigendum to "The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII" [Mol Genet Metab 2020 Mar;129(3):219-227].
Raymond Y Wang , José Francisco da Silva Franco , Jaime López-Valdez , Esmeralda Martins
Molecular Genetics and Metabolism 131 285
2020
Elevated oxysterol and N‐palmitoyl‐O‐phosphocholineserine levels in congenital disorders of glycosylation
An N Dang Do , Irene J Chang , Xutian Jiang , Lynne A Wolfe
Journal of inherited metabolic disease 46 ( 2) 326 -334
3
2023
A concerted action to explore therapies for free sialic acid storage disease
Marjan Huizing , Steven U Walkley , Melissa Wasserstein , Christine Anne-Longin
Molecular Genetics and Metabolism 138 ( 2) 107160 -107160
2023
Intra-Articular AAV9 alpha-Iduronidase Gene Therapy in the Canine Model of Mucopolysaccharidosis Type I Results in Rapid Synovial and Cartilage Iduronidase Expression, Clearance of Heparan Sulfate, and High Serum alpha-Iduronidase Levels
Shih-hsin Kan , Haoyue Zhang , Jodi D Smith , Elizabeth M Snella
MOLECULAR THERAPY 27 ( 4) 435 -436
2019
A long-term observation of ophthalmology assessment in atypical CLN2 patients receiving ICV cerliponase alfa treatment
Audrey Kao , Harriet Chang , Rahul Bhola , Raymond Y Wang
Molecular Genetics and Metabolism 141 ( 2) 107910 -107910
2024
Guidelines on the diagnosis, clinical assessments, treatment and management for CLN2 disease patients.
Sara E Mole , Angela Schulz , Eben Badoe , Samuel F Berkovic
Orphanet Journal of Rare Diseases 16 ( 1) 185 -185
2021
Exploring Pompe disease: Insights into the natural history of novel Gaac. 1826dupA knock-in murine model
Jerry F Harb , Shih-hsin Kan , Nancy D Dalton , Alejandra Padilla
Molecular Genetics and Metabolism 141 ( 2) 107873 -107873
2024