Lysosomal and Rare Disorders Research and Treatment Center

Statistics

Publications

194

Citations

5.8K

Authors

Location

Fairfax, United States

Also known as LDRTC

Lysosomal and Rare Disorders Research and Treatment Center is a other institution in Fairfax, United States known for gaucher disease, life sciences, and medicine.

Homepage ROR

Top concepts

Gaucher DiseaseLife SciencesMedicineEnzyme Replacement TherapyEnzymologyBiochemistryLysosomal Enzyme DeficiencyNeurodegenerative DisordersNeurologyNeurogenetics

Publications

194

Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

Jordi Díaz‐Manera, Priya S. Kishnani, Hani Kushlaf et al. · The Lancet Neurology · 2021 · 184 citations · Full text

Veterinary Medicine, Internal Medicine, Pharmacotherapy +3

A Pilot Study of Omalizumab in Eosinophilic Esophagitis

Denise Loizou, Benjamin Enav, Edina Komlódi-Pásztor et al. · PLoS ONE · 2015 · 165 citations · Full text

Life Sciences, Immunology, Internal Medicine +13

Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1‐year Phase 1/2 clinical trial

Raphael Schiffmann, Özlem Göker-Alpan, Myrl Holida et al. · Journal of Inherited Metabolic Disease · 2019 · 137 citations · Full text

Medicine, Pharmacology, Pharmacotherapy +15

The definition of neuronopathic Gaucher disease

Raphael Schiffmann, Jeff Sevigny, Arndt Rolfs et al. · Journal of Inherited Metabolic Disease · 2020 · 103 citations · Full text

Medicine, Neurology, Pathology +11

Demographics and patient characteristics of 1209 patients with Gaucher disease: Descriptive analysis from the Gaucher Outcome Survey (GOS)

Ari Zimran, Nadia Belmatoug, Bruno Bembi et al. · American Journal of Hematology · 2017 · 94 citations · Full text

Life Sciences, Endocrinology, Pharmacotherapy +10

1–5 of 194

Authors

ÖG
Özlem Göker-Alpan
Lysosomal and Rare Disorders Research and Treatment Center, United States
262 publications · 6.3K citations
41
h-index
RP
Renuka P. Limgala
Lysosomal and Rare Disorders Research and Treatment Center, United States
33 publications · 422 citations
10
h-index
GC
Gérson Carvalho
Lysosomal and Rare Disorders Research and Treatment Center, United States
28 publications · 431 citations
7
h-index
JD
Julia Dao
Lysosomal and Rare Disorders Research and Treatment Center, United States
29 publications · 118 citations
7
h-index
CI
Chidima Ioanou
Lysosomal and Rare Disorders Research and Treatment Center, United States
13 publications · 116 citations
6
h-index

1–5 of 22