Farber disease: design of the first observational and cross-sectional cohort study capturing retrospective and prospective data on the natural history and phenotypic spectrum of patients, including novel methodologies for assessment of disease-specific symptoms

6533b85cfe1ef96bd12bd279

RESEARCH PRODUCT

Farber disease: design of the first observational and cross-sectional cohort study capturing retrospective and prospective data on the natural history and phenotypic spectrum of patients, including novel methodologies for assessment of disease-specific symptoms

Alexander Solyom Boris Hügle John J. Mitchell Edward H. Schuchman Michael Beck

subject

0301 basic medicine Disease specific Pediatrics medicine.medical_specialty Farber disease business.industry Endocrinology Diabetes and Metabolism Prospective data 030105 genetics & heredity medicine.disease Biochemistry Natural history 03 medical and health sciences 0302 clinical medicine Endocrinology Genetics medicine Observational study business Molecular Biology 030217 neurology & neurosurgery Cohort study

year	journal	country	edition	language
2017-01-01	Molecular Genetics and Metabolism

https://doi.org/10.1016/j.ymgme.2016.11.326

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