Ampliação do transplante de células- tronco hematopoiéticas para mucopolissacaridose tipo I
Brasil. Ministério da Saúde. Secretaria de Ciência, Tecnologia e Insumos Estratégicos.
Brasília; CONITEC; jan. 2018.
Não convencional em Português | LILACS, BRISA/RedTESA | ID: biblio-905582
Report of 5 novel mutations of the α-L-iduronidase gene and comparison of Korean mutations in relation with those of Japan or China in patients with mucopolysaccharidosis I.
Clinical and Molecular Characterization of Patients with Mucopolysaccharidosis Type I in an Algerian Series.
Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I.
Neurocognitive and neuropsychiatric phenotypes associated with the mutation L238Q of the α-L-iduronidase gene in Hurler-Scheie syndrome.
Insights into mucopolysaccharidosis I from the structure and action of α-L-iduronidase.
12 year follow up of enzyme-replacement therapy in two siblings with attenuated mucopolysaccharidosis I: the important role of early treatment.
Engineering a lysosomal enzyme with a derivative of receptor-binding domain of apoE enables delivery across the blood-brain barrier.
The natural history of MPS I: global perspectives from the MPS I Registry.
Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry.
Airway-related symptoms and surgeries in patients with mucopolysaccharidosis I.