Results 21 to 30 of about 793,688 (114)
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
American Journal of Medical Genetics Part A, Volume 185, Issue 11, Page 3350-3358, November 2021., 2021 Abstract From Sir Archibald Garrod's initial description of the tetrad of albinism, alkaptonuria, cystinuria, and pentosuria to today, the field of medicine dedicated to inborn errors of metabolism has evolved from disease identification and mechanistic discovery to the development of therapies designed to subvert biochemical defects.
Hilary J. Vernon, Irini Manoli
wiley +1 more source
Internal Medicine Journal, Volume 51, Issue 10, Page 1580-1593, October 2021., 2021 Abstract This study aims to evaluate the impact of the COVID‐19 pandemic on the lysosomal disorders unit (LSDU) at Royal Free London NHS Foundation Trust (RFL), a highly specialised national service for diagnosis and management of adults with lysosomal storage disorders (LSD). Review of home care enzyme replacement therapy (ERT) and emergency care, and
Uma Ramaswami +6 more
wiley +1 more source
Oral adverse effects: drug‐induced tongue disorders
Oral Diseases, Volume 27, Issue 6, Page 1528-1541, September 2021., 2021 Abstract Objectives Due to a worldwide increase in drug consumption, oral healthcare professionals are frequently confronted with patients using one or more drugs. A large number of drugs can be accompanied with adverse drug reactions in the orofacial region, amongst others of the tongue.
Yalda Aziz +7 more
wiley +1 more source
BMC Medical Genomics, 2021 Background This article presents the results of long-term observations and comparative analysis of genotype–phenotype features in a large group of patients (227 males and one female) with a severe, intermediate and mild form of Hunter syndrome ...
Alla Nikolaevna Semyachkina +3 more
doaj +1 more source
Corrigendum to: Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study [PDF]
, 2023 Corrigendum to: Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized ...
Jones, Simon A. +14 more
core +1 more source
Corrigendum to “Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II” [PDF]
, 2023 Corrigendum to “Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis ...
Jones, Simon A. +14 more
core +1 more source
Active immunotherapy and alternative therapeutic modalities for Alzheimer's disease
Alzheimer's &Dementia: Translational Research &Clinical Interventions, Volume 6, Issue 1, 2020., 2020 Abstract As knowledge of Alzheimer's disease (AD) progression improves, the field has recognized the need to diversify the pipeline, broaden strategies and approaches to therapies, as well as delivery mechanisms. A better understanding of the earliest biological processes of AD/dementia would help inform drug target selection.
Stacie Weninger +12 more
wiley +1 more source
BioMed Research International, Volume 2020, Issue 1, 2020., 2020 Purpose. Mucopolysaccharidoses (MPS) are group of inherited lysosomal storage diseases caused by mutations of enzymes involved in catalyzing different glycosaminoglycans (GAGs). MPS I and MPS II exhibit both somatic and neurological symptoms with a relatively high disease incidence.
Haiyan Nan +3 more
wiley +1 more source