J
Joel Charrow
Researcher at Northwestern University
Publications - 167
Citations - 8782
Joel Charrow is an academic researcher from Northwestern University. The author has contributed to research in topics: Enzyme replacement therapy & Neurofibromatosis. The author has an hindex of 46, co-authored 151 publications receiving 7767 citations. Previous affiliations of Joel Charrow include Children's Memorial Hospital.
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Journal ArticleDOI
Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry
Neal J. Weinreb,Joel Charrow,Hans C. Andersson,Paige Kaplan,Edwin H. Kolodny,Pramod K. Mistry,Gregory M. Pastores,Barry E. Rosenbloom,C. Ronald Scott,Rebecca S. Wappner,Ari Zimran +10 more
TL;DR: Enzyme replacement therapy prevents progressive manifestations of Gaucher disease, and ameliorates Gaucher Disease-associated anemia, thrombocytopenia, organomegaly, bone pain, and bone crises.
Journal ArticleDOI
The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease.
Joel Charrow,Hans C. Andersson,Paige Kaplan,Edwin H. Kolodny,Pramod K. Mistry,Gregory M. Pastores,Barry E. Rosenbloom,C. Ronald Scott,Rebecca S. Wappner,Neal J. Weinreb,Ari Zimran +10 more
TL;DR: The Registry will be useful in evaluating the effects of specific therapies in GD and the possible influences of environment, ethnicity, and genotype on the natural history of the disorder.
Journal ArticleDOI
Natural history of optic pathway tumors in children with neurofibromatosis type 1: a longitudinal study.
TL;DR: It is concluded that OPT rarely progress during the next few years in children with neurofibromatosis type 1 once the OPT have been discovered and the utility of screening neuroimaging for OPT in symptom-free children with NF-1 appears very limited.
Journal ArticleDOI
Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with Fabry disease.
Dominique P. Germain,Stephen Waldek,Maryam Banikazemi,Maryam Banikazemi,David A. Bushinsky,Joel Charrow,Robert J. Desnick,Philip J. Lee,Thomas W. Loew,Anouk C. Vedder,Rekha Abichandani,William R. Wilcox,Nathalie Guffon +12 more
TL;DR: Long-term agalsidase beta therapy stabilizes renal function in patients without renal involvement at baseline, maintains reduction of plasma GL-3, and sustainsGL-3 clearance in capillary endothelial cells and multiple renal cell types.
Journal ArticleDOI
Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat
Dominique P. Germain,Derralynn Hughes,Kathleen Nicholls,Daniel G. Bichet,Roberto Giugliani,William R. Wilcox,Claudio Feliciani,Suma P. Shankar,Fatih Süheyl Ezgü,Hernan Amartino,Drago Bratkovic,Ulla Feldt-Rasmussen,Khan Nedd,Usama A Sharaf El Din,Charles Marques Lourenço,Maryam Banikazemi,Joel Charrow,Majed Dasouki,David N. Finegold,P Giraldo,Ozlem Goker-Alpan,Nicola Longo,C. Ronald Scott,Roser Torra,Ahmad Tuffaha,Ana Jovanovic,Stephen Waldek,Seymour Packman,Elizabeth Ludington,Christopher Viereck,John Kirk,Julie Yu,Elfrida R. Benjamin,Franklin K. Johnson,David J. Lockhart,Nina Skuban,Jeff Castelli,Jay A. Barth,Carrolee Barlow,Raphael Schiffmann,Raphael Schiffmann +40 more
TL;DR: Among all randomly assigned patients with Fabry's disease (with mutant α-galactosidase forms that were suitable or not suitable for migalastat therapy), the percentage of patients who had a response at 6 months did not differ significantly between the migAlastat group and the placebo group.