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http://dx.doi.org/10.1016/j.ambp.2006.11.006 | DOI Listing |
Clin Trials
January 2025
Rare Diseases Team, Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD, USA.
Background/aims: Rare disease drug development faces unique challenges, such as genotypic and phenotypic heterogeneity within small patient populations and a lack of established outcome measures for conditions without previously successful drug development programs. These challenges complicate the process of selecting the appropriate trial endpoints and conducting clinical trials in rare diseases. In this descriptive study, we examined novel drug approvals for non-oncologic rare diseases by the U.
View Article and Find Full Text PDFFront Biosci (Landmark Ed)
January 2025
Division of Molecular Psychiatry, Center of Mental Health, University of Hospital Würzburg, 97080 Würzburg, Germany.
Background: The inheritance of the short allele, encoding the serotonin transporter (SERT) in humans, increases susceptibility to neuropsychiatric and metabolic disorders, with aging and female sex further exacerbating these conditions. Both central and peripheral mechanisms of the compromised serotonin (5-HT) system play crucial roles in this context. Previous studies on SERT-deficient (Sert) mice, which model human SERT deficiency, have demonstrated emotional and metabolic disturbances, exacerbated by exposure to a high-fat Western diet (WD).
View Article and Find Full Text PDFEur Stroke J
January 2025
Department of Neurology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Background: We aimed to assess impairments on health-related quality of life, and mental health resulting from Retinal artery occlusion (RAO) with monocular visual field loss and posterior circulation ischemic stroke (PCIS) with full or partial hemianopia using patient-reported outcome measures (PROMs).
Methods: In a prospective study, consecutive patients with acute RAO on fundoscopy and PCIS on imaging were recruited during their surveillance on a stroke unit over a period of 15 months. Baseline characteristics were determined from medical records and interviews.
Nord J Psychiatry
January 2025
Mental Health Center Copenhagen, Copenhagen University Hospital, Copenhagen, Denmark.
Purpose: People living in supported accommodation often have complex care needs, including longer-term mental health illness and physical health comorbidities. Effective coordination between health and supported accommodation services is crucial to address these needs. However, evidence on the effectiveness of healthcare interventions in this setting remains limited.
View Article and Find Full Text PDFDisabil Rehabil
January 2025
Sydney School of Health Sciences, Faculty of Medicine & Health, The University of Sydney, Sydney, Australia.
Purpose: To investigate potential mechanisms of a digital rehabilitation intervention associated with improved mobility among adults undertaking rehabilitation.
Materials And Methods: Causal mediation analysis of the AMOUNT trial (ACTRN12614000936628). Participants were randomised to digitally-enabled rehabilitation (virtual reality video games, activity monitors, and handheld computer devices prescribed by a physiotherapist) and usual care or usual care alone.
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