Results 161 to 170 of about 463,913 (214)
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Onasemnogene abeparvovec (OA) is an AAV9‐based gene therapy for spinal muscular atrophy type I (SMA I). Real‐world outcomes show increased response variability compared to clinical trials, and follow‐up data beyond 12–18 months are limited.
Marika Pane +43 more
wiley +1 more source
Clinical Validation of Plasma p‐217tau in Neurological Diseases
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Plasma p‐217tau is a minimally invasive but specific biomarker for diagnosing Alzheimer's disease (AD). However, its disease specificity remains to be clinically evaluated. We validated the reliability of the p‐217tau biomarker in 12 other neurological diseases.
Takeshi Kawarabayashi +13 more
wiley +1 more source
An Ethically Appropriate Response to Individuals with Gender Dysphoria [PDF]
, 2020 Lindevaldsen, Rena M
core +1 more source
Arthritis Care &Research, EarlyView.Objective A leading cause of death among patients with scleroderma (SSc), interstitial lung disease (ILD) remains challenging to prognosticate. The discovery of biomarkers that accurately determine which patients would benefit from close monitoring and aggressive therapy would be an essential clinical tool.
Cristina M. Padilla +13 more
wiley +1 more source
Arthritis Care &Research, EarlyView.Objective The objective was to identify factors determining acute arthritis resolution and safety with colchicine and prednisone in acute calcium pyrophosphate (CPP) crystal arthritis. Methods We conducted a post hoc analysis of the COLCHICORT trial, which compared colchicine and prednisone for the treatment of acute CPP crystal arthritis, using a ...
Tristan Pascart +14 more
wiley +1 more source
Arthritis Care &Research, EarlyView.Objective To evaluate the efficacy and safety of baricitinib in pediatric patients with active juvenile idiopathic arthritis–associated uveitis (JIA‐U) or chronic anterior antinuclear antibody–positive uveitis, who had an inadequate response to methotrexate (MTX) or biologic disease‐modifying antirheumatic drugs (bDMARDs).
Athimalaipet V. Ramanan +7 more
wiley +1 more source
Financial Distress and Its Determinants in Rheumatoid Arthritis
Arthritis Care &Research, EarlyView.Objective To quantify the degree of financial distress and identify its determinants in adults with rheumatoid arthritis (RA) given the frequent prolonged use of expensive disease‐modifying therapies. Methods We identified adults enrolled in the FORWARD databank with either RA or noninflammatory musculoskeletal disease (NIMSKD) completing the ...
Amber Brown Keebler +5 more
wiley +1 more source
Arthritis Care &Research, EarlyView.Objective Antinuclear antibodies (ANAs) are present at high titers in 2% of the general population, but their clinical significance in individuals without an autoimmune (AI) disease is not known. We tested the hypothesis that the presence of a high ANA titer in non‐AI conditions is associated with disease.
Matthew Chung +7 more
wiley +1 more source
Arthritis Care &Research, EarlyView.Objective This study aimed to characterize the pharmacokinetics, pharmacodynamics, safety, and exploratory efficacy of subcutaneous belimumab in pediatric patients with active systemic lupus erythematosus (SLE) receiving standard therapy. Methods This single‐arm, multicenter, open‐label trial (GSK study 200908; ClinicalTrials.gov identifier ...
Hermine I. Brunner +14 more
wiley +1 more source
Arthritis Care &Research, EarlyView.Objective Sjögren's disease is an autoimmune disorder that can impact multiple organ systems, including the peripheral nervous system (PNS). PNS manifestations, which can exist concurrently, include mononeuropathies, polyneuropathies, and autonomic nervous system neuropathies.
Anahita Deboo +88 more
wiley +1 more source