Easy-to-use algorithm would provide faster diagnoses for mucopolysaccharidosis type I and enable patients to receive earlier treatment
نویسندگان
چکیده
منابع مشابه
Willingness to Receive Treatment for Hepatitis C among Injecting Drug Users on Methadone Program: Implications for Education and Treatment
Background: Hepatitis C virus (HCV) is common among people who inject drugs (PWID) on methadone program in Iran (Persia). However, a few PWID on methadone program report willingness to receive HCV treatment. This study aimed to assess the factors which were associated with willingness to receive HCV treatment in a group of PWID on methadone program in Iran.Methods: We surveyed 187 PWID at seven...
متن کاملExperiences of parents and patients with the timing of Mucopolysaccharidosis type I (MPS I) diagnoses and its relevance to the ethical debate on newborn screening.
INTRODUCTION Newborn screening (NBS) techniques have been developed for several lysosomal storage disorders (LSDs), including Mucopolysaccharidosis type I (MPS I). MPS I is an LSD with a wide phenotypic spectrum that ranges from the severe Hurler phenotype to the attenuated Scheie phenotype. To improve the ethical discussion about NBS for MPS I, we performed an interview study to explore the ex...
متن کاملErratum to: "Willingness to Receive Treatment for Hepatitis C among Injecting Drug Users on Methadone Program: Implications for Education and Treatment"
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متن کاملAn algorithm to predict phenotypic severity in mucopolysaccharidosis type I in the first month of life
INTRODUCTION Mucopolysaccharidosis type I (MPS I) is a progressive multisystem lysosomal storage disease caused by deficiency of the enzyme α-L-iduronidase (IDUA). Patients present with a continuous spectrum of disease severity, and the most severely affected patients (Hurler phenotype; MPS I-H) develop progressive cognitive impairment. The treatment of choice for MPS I-H patients is haematopoi...
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ژورنال
عنوان ژورنال: Acta Paediatrica
سال: 2018
ISSN: 0803-5253
DOI: 10.1111/apa.14417