Results 1 to 10 of about 50 (48)
Molecular Genetics and Metabolism Reports, 2023 Mucopolysaccharidosis (MPS) type II (Hunter syndrome) is a rare X-linked, recessive, lysosomal storage disorder caused by the deficit of the enzyme iduronate 2-sulfatase (IDS), resulting in accumulation of glycosaminoglycans (GAGs) impairing cellular ...
Mei-Yan Chan +2 more
doaj +3 more sources
Molecular Genetics and Metabolism Reports, 2017 Mucopolysaccharidosis (MPS) II or Hunter syndrome is a chronic, progressive, multi-systemic illness associated with significant morbidity and early mortality. Available evidence in Asian populations shows that Hunter syndrome has a mean age of onset of 2
Lock-Hock Ngu +3 more
doaj +3 more sources
Molecular Therapy: Methods & Clinical Development, 2021 This open-label, phase 1/2 study (JMACCT CTR JMA-IIA00350) evaluated the efficacy and safety of intracerebroventricular idursulfase beta in patients with mucopolysaccharidosis II (MPS II). Herein, we report the 100-week results.
Joo-Hyun Seo +4 more
doaj +1 more source
Педиатрическая фармакология, 2022 Background. Enzyme replacement therapy (ERT) with iduronate-2-sulfatase recombinant forms (idursulfase and idursulfase beta) is effective for the management of mucopolysaccharidosis type II (MPS II).
Julia G. Levina +9 more
doaj +1 more source
Idursulfase Beta (Hunterase) Therapeutic Experience in a Patient with Mucopolysaccharidosis Type II
Вопросы современной педиатрии, 2023 Background. Mucopolysaccharidosis type II (MPS II, Hunter syndrome) is an X-linked recessive disease caused by lysosomal enzyme iduronate-2-sulfatase deficiency resulting in progressive glycosaminoglycans (GAG) accumulation in tissues (dermatan sulfate ...
Nato D. Vashakmadze +4 more
doaj +1 more source
Dose selection for biological enzyme replacement therapy indicated for inborn errors of metabolism
Clinical and Translational Science, Volume 16, Issue 12, Page 2438-2457, December 2023., 2023 Abstract This paper summarizes key features of the dose‐finding strategies used in the development of 11 approved new molecular entities that are first‐in‐class enzyme replacement therapy (ERT), with a goal to gain insight into the dose exploration approaches to inform efficient dose‐finding in future development of biological products for Inborn ...
Yuen Yi Hon +8 more
wiley +1 more source
Allergic Reactions at Enzyme Replacement Therapy in Children with Mucopolysaccharidosis Type II
Вопросы современной педиатрии, 2021 Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is rare hereditary disease caused by changes in the IDS gene and associated deficiency of lysosomal enzyme iduronate-2-sulfatase (I2S).
Julia G. Levina +7 more
doaj +1 more source
Advances in therapies for neurological lysosomal storage disorders
Journal of Inherited Metabolic Disease, Volume 46, Issue 5, Page 874-905, September 2023., 2023 Abstract Lysosomal Storage Disorders (LSDs) are a diverse group of inherited, monogenic diseases caused by functional defects in specific lysosomal proteins. The lysosome is a cellular organelle that plays a critical role in catabolism of waste products and recycling of macromolecules in the body.
S. Ellison, H. Parker, B. Bigger
wiley +1 more source
Accelerated approval drug labels often lack information for clinical decision‐making
Pharmacotherapy: The Journal of Human Pharmacology and Drug Therapy, Volume 43, Issue 4, Page 300-304, April 2023., 2023 Abstract Study objective We evaluated US Food and Drug Administration labels for drugs approved under the accelerated approval pathway and whether these labels contained in sufficient information regarding their accelerated approval. Design Retrospective, observational, cohort study.
Jeromie Ballreich +5 more
wiley +1 more source
The Inflation Reduction Act: A boon for the generic and biosimilar industry
Journal of Clinical Pharmacy and Therapeutics, Volume 47, Issue 11, Page 1738-1751, November 2022., 2022 The Inflation Reduction Act gives a significant boost to generics and biosimilars!Soon lowered out‐of‐pocket costBiosimilars promoted and adopted Abstract What is Known and Objective After more than a year of negotiation, a $740 billion climate and health care bill known as The Inflation Reduction Act (IRA) became law on 16 August 2022.
Sarfaraz K. Niazi
wiley +1 more source