Frequency of iduronate-2-sulfatase gene variants detected in newborn screening for mucopolysaccharidosis type II in Japan. [PDF]
Hattori Y +8 more
europepmc +1 more source
Trends in orphan drug use and spending among children and adolescents during 2010-2020 in Korea. [PDF]
Oh JY, Lee JY, Kim DS.
europepmc +1 more source
Profile of idursulfase for the treatment of Hunter syndrome [PDF]
Daniela Concolino +5 more
core +2 more sources
Brain Cancer Chemotherapy through a Delivery System across the Blood-Brain Barrier into the Brain Based on Receptor-Mediated Transcytosis Using Monoclonal Antibody Conjugates. [PDF]
Tashima T.
europepmc +1 more source
Heparan sulfate in cerebrospinal fluid as a biomarker to assess disease severity and for treatment monitoring in patients with Mucopolysaccharidosis Type II: a position statement. [PDF]
Giugliani R +7 more
europepmc +1 more source
How to manage hypersensitivity reactions to enzyme replacement therapy in lysosomal storage diseases? [PDF]
Spataro F +5 more
europepmc +1 more source
Pharmacotherapeutic Patterns and Patients' Access to Pharmacotherapy for Some Rare Diseases in Bulgaria - A Pilot Comparative Study. [PDF]
Kamusheva M +4 more
europepmc +1 more source
Intravenous Idursulfase for the Treatment of Mucopolysaccharidosis Type II: A Systematic Literature Review. [PDF]
Al-Hertani W +6 more
europepmc +1 more source
Advancing the Research and Development of Enzyme Replacement Therapies for Lysosomal Storage Diseases. [PDF]
Puhl AC, Ekins S.
europepmc +1 more source
Characterization of Treatment Utilization in a Metabolic Genetics Clinic. [PDF]
Riedy M, Briggs J, Huang T.
europepmc +1 more source

