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Lysosomal and Rare Disorders Research and Treatment Center | Researchclopedia

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Lysosomal and Rare Disorders Research and Treatment Center

Fairfax, Virginiaother

158

Works

5,039

Citations

35

h-index

Top Research Topics

Lysosomal Storage Disorders Research Carbohydrate Chemistry and Synthesis Cellular transport and secretion

Top Researchers

Eiki Kominami Juntendo University

382 works53,391 citationsh-index: 87

Recent Research

Improvement of Bone Mineral Density in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: Results from Clinical Studies

Ari Zimran,Jean Botha,et al.

20260 citations

Trypanosoma species research and implications

Glycogen Storage Diseases and Myoclonus

Studies on Chitinases and Chitosanases

Glycosylation and Glycoproteins Research

Child Nutrition and Feeding Issues

Calcium signaling and nucleotide metabolism

Autophagy in Disease and Therapy

Galectins and Cancer Biology

Biochemical and Molecular Research

Giancarlo Parenti Department of Health

322 works19,252 citationsh-index: 55

Derralynn Hughes Royal Free London NHS Foundation Trust

663 works18,357 citationsh-index: 74

Atul Mehta Mount Sinai Health System

376 works16,512 citationsh-index: 70

Cynthia K. Thompson Northwestern University

332 works14,055 citationsh-index: 69

Wim J. Sluiter University Medical Center Groningen

309 works12,942 citationsh-index: 64

Long-term efficacy and safety of pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease: results from up to 5 years of the BRIGHT F51 phase III, open-label extension study

Myrl Holida,Aleš Linhart,et al.

20260 citations

Maternal and Fetal Outcomes in Imiglucerase‐Treated Patients With Gaucher Disease: Real‐World Evidence From the International Collaborative Gaucher Group ( <scp>ICGG</scp> ) Gaucher Registry Pregnancy Sub‐Registry

Shoshana Revel‐Vilk,P B Deegan,et al.

20260 citations

Clinical expert opinion on the role of elosulfase alfa in non-ambulatory individuals with Morquio A syndrome

Carolina FM De Souza,Barbara K. Burton,et al.

20260 citations

Lucerastat, an oral therapy for Fabry disease: results from a pivotal randomized phase 3 study and its open-label extension

Peter Nordbeck,Ozlem Goker-Alpan,et al.

20260 citations

P169: 90-month efficacy outcomes with cipaglucosidase alfa plus miglustat in adults with Pompe disease in ATB200-02: An open-label phase I/II study

Priya Kishnani,Drago Bratkovic,et al.

20260 citations