Publications by authors named "Eric Crombez"

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Gene therapy for hemophilia B: results from the phase 1/2 101HEMB01/02 studies.
Steven Pipe, Allen Poma, Anita Rajasekhar, Tamara Everington, Serap Sankoh, Eric Crombez

Blood Adv· June 2025

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Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa).
David A Weinstein, Terry G Derks, David F Rodriguez-Buritica, Ayesha Ahmad, María-Luz Couce, Eric Crombez

J Inherit Metab Dis· March 2025

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Metachromatic Leukodystrophy: An Assessment of Disease Burden.
Florian S Eichler, Timothy M Cox, Eric Crombez, Christine Í Dali, Alfried Kohlschütter

J Child Neurol· November 2016

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Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naïve to enzyme replacement therapy or previously treated with imiglucerase.
Laurie Smith, William Rhead, Joel Charrow, Suma P Shankar, Ashish Bavdekar, Eric Crombez

Mol Genet Metab· February 2016

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Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher disease.
Deborah Elstein, Atul Mehta, Derralynn A Hughes, Pilar Giraldo, Joel Charrow, Eric Crombez

Am J Hematol· July 2015

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Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long-term data from phase III clinical trials.
Derralynn A Hughes, Derlis E Gonzalez, Elena A Lukina, Atul Mehta, Madhulika Kabra, Eric Crombez

Am J Hematol· July 2015

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Seven-year safety and efficacy with velaglucerase alfa for treatment-naïve adult patients with type 1 Gaucher disease.
Ari Zimran, Nan Wang, Carol Ogg, Eric Crombez, Gabriel M Cohn

Am J Hematol· July 2015

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Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase.
Ari Zimran, Gregory M Pastores, Anna Tylki-Szymanska, Derralynn A Hughes, Deborah Elstein, Eric Crombez

Am J Hematol· March 2013

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Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double-blind, multinational, Phase 3 study.
Derlis E Gonzalez, Hadhami Ben Turkia, Elena A Lukina, Isaac Kisinovsky, Marie-Françoise Ben Dridi, Eric Crombez

Am J Hematol· March 2013

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Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease.
Hadhami Ben Turkia, Derlis E Gonzalez, Norman W Barton, Ari Zimran, Madhulika Kabra, Eric Crombez

Am J Hematol· March 2013

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Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled study.
Friedrich K Trefz, Barbara K Burton, Nicola Longo, Mercedes Martinez-Pardo Casanova, Daniel J Gruskin, Eric A Crombez

J Pediatr· May 2009

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Clinical and molecular heterogeneity in patients with the cblD inborn error of cobalamin metabolism.
Isabelle R Miousse, David Watkins, David Coelho, Tony Rupar, Eric A Crombez

J Pediatr· April 2009

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Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria.
Phillip Lee, Eileen P Treacy, Eric Crombez, Melissa Wasserstein, Lewis Waber

Am J Med Genet A· November 2008

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Maternal glutaric acidemia, type I identified by newborn screening.
Eric A Crombez, Stephen D Cederbaum, Elaine Spector, Erica Chan, Denise Salazar

Mol Genet Metab· May 2008

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Expanded newborn screening identifies maternal primary carnitine deficiency.
Lisa A Schimmenti, Eric A Crombez, Bernd C Schwahn, Bryce A Heese, Timothy C Wood

Mol Genet Metab· April 2007

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Duplication of the Down syndrome critical region does not predict facial phenotype in a baby with a ring chromosome 21.
Eric A Crombez, Katrina M Dipple, Lisa A Schimmenti, Nagesh Rao

Clin Dysmorphol· October 2005

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Pitfalls in newborn screening.
Eric Crombez, Richard Koch, Stephen Cederbaum

J Pediatr· July 2005

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Hyperargininemia due to liver arginase deficiency.
Eric A Crombez, Stephen D Cederbaum

Mol Genet Metab· March 2005

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