Results 241 to 250 of about 3,558,519 (308)

Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective There are limited real‐world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one‐time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real‐world outcomes for OA in Japan.
Kayoko Saito, Kamal Benguerba, Ken Tsuchida, Kazushige Yazawa, Isao Tsumiyama, Hiromitsu Kayama, Sandra P. Reyna, Farid Khan, Richard S. Finkel   +8 more
wiley   +1 more source

Racial and Geographic Disparities in Automated External Defibrillator Use During EMS Encounters in the United States. [PDF]

Healthcare (Basel)
Kreysa PG.
europepmc   +1 more source

Prominent Movement Disorders in RNU2‐2‐Related Spliceosomopathy

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Pediatric movement disorders often overlap with neurodevelopmental diseases, suggesting shared molecular mechanisms. Variants in small nuclear RNA (snRNA) genes encoding spliceosome components have recently been associated with neurodevelopmental disorders, termed “RNUopathies.” We analyzed genome sequencing data from 14 patients with ...
Magdalena Krygier, Ugo Sorrentino, Matias Wagner, Marta Zawadzka, Anna Lemska, Maria Mazurkiewicz‐Bełdzińska, Michael Zech   +6 more
wiley   +1 more source

Polysubstance use in adolescents: conceptual issues and the need for a new public health framework and approach. [PDF]

Psychol Med
Fleury R, Staines L, Smyth B, Cannon M.
europepmc   +1 more source

T1 Over Squared Proton Density Ratio to Characterize Multiple Sclerosis Lesions

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Differentiating remyelinated from demyelinated lesions in MS remains challenging without histological confirmation. This study introduces the T1‐to‐PD2 ratio (TPR) imaging approach and evaluates its ability to characterize MS lesions alongside other quantitative MRI (qMRI) metrics. Methods Thirty individuals with MS (mean age: 47.5 ± 
Sarah J. Wright, Scotty G. McKay, Jean Ramos Bou, Fen Bao, Vivian B. Truong, Chenyang Li, Li Jiang, Anza B. Memon, E. Mark Haacke, Yulin Ge, Yongsheng Chen   +10 more
wiley   +1 more source

Compiling Comprehensive Databases for Opioid Overdose Monitoring in a Fragmented Health Care System. [PDF]

Health Serv Res
Hodgkin D, Green TC.
europepmc   +1 more source

Assessing the levels of utilisation of WHO-recommended healthcare waste disposal practices at healthcare service delivery points in Uganda. [PDF]

PLOS Glob Public Health
Senkubuge S, Kabwijamu L, Nabukeera S, Tusubira AK, Kasule T, Makumbi FE, Etajak S, Kayemba CN.   +7 more
europepmc   +1 more source

Retraction: HIV/AIDS in Indonesia: current treatment landscape, future therapeutic horizons, and herbal approaches. [PDF]

Front Public Health
Frontiers Editorial Office.
europepmc   +1 more source

From authorisation to clinical practice: evolution of the use of biological medicines according to the SmPC and guidelines (2006 to 2025). [PDF]

Eur J Clin Pharmacol
Penninx BMF, Hollak CEM, Tas SW, Timmers L, de Visser SJ, van Kempen ZLE.   +5 more
europepmc   +1 more source