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ISSN 2208-150X (Print)

ISSN 2208-1518 (Online)

THE BEST OF ICIEM 2017

RNA Sequencing Helps in the Genetic Diagnosis of Metabolic Disorders • Newborn Screening Programs

Should Be Aware of a Rare SNP in the Placental Riboflavin Transporter Gene • A Novel Signaling Pathway May

Mediate Cholesterol Homeostasis in Niemann-Pick Type C Disease • Experience With Hematopoietic Stem Cell

Transplantation for Mucopolysaccharidosis Type 1H

The production and distribution of this publication is sponsored by Sanofi Genzyme

13TH INTERNATIONAL CONGRESS

OF INBORN ERRORS OF

METABOLISM (ICIEM)

5–8 SEPTEMBER 2017

RIO DE JANEIRO • BRAZIL