Results 21 to 30 of about 550 (133)

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, Mariana Socal, Charles L. Bennett, Andrew Xuan, Antonio Trujillo, Gerard Anderson   +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

Milestones in treatments for inborn errors of metabolism: Reflections on Where chemistry and medicine meet

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

Impact of SARS‐CoV‐2 (COVID‐19) pandemic on patients with lysosomal storage disorders and restoration of services: experience from a specialist centre

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, Simona D'Amore, Niamh Finnegan, Derralynn Hughes, Masoud Kazemi, Lysosomal Disorders Team, Royal Free London NHS Foundation Trust   +6 more
wiley   +1 more source

Analysis of long-term observations of the large group of Russian patients with Hunter syndrome (mucopolysaccharidosis type II)

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, Elena Yurievna Voskoboeva, Ekaterina Alexandrovna Nikolaeva, Ekaterina Yurievna Zakharova   +3 more
doaj   +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, Willem Maria Hubertus Rademacher, Atty Hielema, Scott Bradley Patton Wishaw, Denise Edwina van Diermen, Jan de Lange, Arjan Vissink, Frederik Reinder Rozema   +7 more
wiley   +1 more source

Induced Pluripotent Stem Cells for the Treatment of Lysosomal Storage Disorders. [PDF]

J Inherit Metab Dis
ABSTRACT Lysosomal disorders (LSDs) are a group of rare metabolic disorders, with an overall incidence of 1:4800 to 1:8000 live births. LSDs are primarily caused by dysfunctional lysosomal enzymes, which typically lead to the progressive accumulation of substrates within cellular lysosomes.
Lorino M, Qiu B, Bigger B.
europepmc   +2 more sources

Mucopolysaccharidoses and the Blood-brain Barrier [PDF]

, 2022
Mucopolysaccharidoses comprise a set of genetic diseases marked by an enzymatic dysfunction in the degradation of glycosaminoglycans in lysosomes. There are eight clinically distinct types of mucopolysaccharidosis, some with various subtypes, based on ...
Goodman, Grant W, Li, Jun, Sahin, Onur, Thompson, Hannah P, Urayama, Akihiko   +4 more
core   +2 more sources

Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act [PDF]

, 2015
Background: Lysosomal storage disorders are a heterogeneous group of approximately 50 monogenically inherited orphan conditions. A defect leads to the storage of complex molecules in the lysosome, and patients develop a complex multisystemic phenotype of
Hoffmann, Georg Friedrich, Mechler, Konstantin, Mountford, William K., Ries, Markus   +3 more
core   +1 more source

Enzyme replacement therapy for the treatment of Hunter disease: A systematic review with narrative synthesis and meta-analysis [PDF]

, 2020
Background: In the last 10 years enzyme replacement therapy (ERT) has become an alternative for the treatment of patients with Hunter disease (HD). Nevertheless, the information regarding efficacy and safety is scarce and mainly based on the pivotal ...
Asensio Tomás, Maria Luisa, Bonet, David, Cañizares Navarro, Ruth, Esteban-Giner, María José, Esteve Atienzar, Pedro Jesús, Giner-Galvañ, Vicente, López Amorós, Ana, Peris García, Jorge, Robert, Jaqueline, Seguí-Ripoll, José Miguel, Wikman-Jorgensen, Philip Erick   +10 more
core   +1 more source