A novel tool for mapping disease severity and outcomes in patients with Gaucher disease utilising the therapeutic goals

6533b7d7fe1ef96bd1268dd8

RESEARCH PRODUCT

A novel tool for mapping disease severity and outcomes in patients with Gaucher disease utilising the therapeutic goals

Gregory M. Pastores Eugen Mengel Gil Faran Neal J. Weinreb Pilar Gilrado Bruno Bembi Olaf Bodamer Maria Domenica Cappellini Patrick Deegan Derralynn Hughes Nadia Belmatoug Deborah Elstein Ari Zimran

subject

medicine.medical_specialty Endocrinology Disease severity business.industry Endocrinology Diabetes and Metabolism Genetics medicine In patient Disease Intensive care medicine business Molecular Biology Biochemistry

year	journal	country	edition	language
2011-02-01	Molecular Genetics and Metabolism

https://doi.org/10.1016/j.ymgme.2010.11.154

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