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Rare Disease Therapeutics (United States) | Researchclopedia

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Rare Disease Therapeutics (United States)

Franklin, Tennesseecompany

92

Works

14,567

Citations

37

h-index

Top Research Topics

Muscle Physiology and Disorders Neurogenetic and Muscular Disorders Research Metabolism and Genetic Disorders

Top Researchers

Mitchell M. Levy Brown University

539 works124,359 citationsh-index: 86

Recent Research

Heart Transplantation and Ventricular Assist Device in Duchenne Muscular Dystrophy: A New Era

David N. Rosenthal,A. Amodeo,et al.

20250 citations

CAR-T cell therapy research

Cancer Immunotherapy and Biomarkers

Advanced biosensing and bioanalysis techniques

Viral-associated cancers and disorders

Pituitary Gland Disorders and Treatments

Cardiomyopathy and Myosin Studies

Protein Degradation and Inhibitors

Palliative Care and End-of-Life Issues

Mitochondrial Function and Pathology

Gordon D. Rubenfeld Sunnybrook Health Science Centre

391 works103,554 citationsh-index: 100

Francis J. Giles

1,183 works57,274 citationsh-index: 125

J. Randall Curtis Foundation for Advancement of International Medical Education and Research

893 works47,451 citationsh-index: 118

Richard J. Cook University of Waterloo

925 works40,108 citationsh-index: 103

Troyen A. Brennan Harvard University

421 works31,113 citationsh-index: 81

A Particularly Severe Diarrhea in Young Infants With Klebsiella spp. Cultured From the Stool: A Case Series

Joanna Mańdziuk,Dominika Rykowska,et al.

20250 citations

Exposure-response of serum biomarkers to vamorolone, a dissociative corticosteroidal anti-inflammatory drug, in 4- to <7-year children

Swati Mummidivarpu,Utkarsh J. Dang,et al.

20250 citations

Dystrophinopathy in ACTION: The first 500 males enrolled in the Advanced Cardiac Therapies Improving Outcomes Network prospective dystrophinopathy registry

Emily A. Hayes,Chet Villa,et al.

20250 citations

Pediatric idiopathic multicentric castleman disease is often severe and responds to siltuximab

Bridget Austin,Saishravan Shyamsundar,et al.

20250 citations

Comparable response rates to therapies in idiopathic multicentric castleman disease patients with and without expert-confirmed diagnoses

Larissa Borys,Bridget Austin,et al.

20250 citations