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MOG antibody-associated disease epidemiology in Olmsted County, USA, and Martinique.
J Neurol
January 2025
Department of Neurology, Mayo Clinic, Rochester, MN, USA.
Objectives: To report myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) epidemiology in two American regions using 2023 diagnostic criteria.
Patients And Methods: We compared age- and sex-adjusted incidence and prevalence of MOGAD per 2023 diagnostic criteria in Olmsted County (Minnesota [USA]) and Martinique (Caribbean [FR]) (01/01/2003-12/31/2018, prevalence day) using Poisson regression. Archived sera in 68-85% were available for MOG-IgG testing by live cell-based assay at Mayo Clinic.
Content Validity of the Friedreich Ataxia Rating Scale in Patients with Spinocerebellar Ataxia.
Neurol Ther
January 2025
Biohaven Pharmaceuticals, Inc., 215 Church Street, New Haven, CT, 06510, USA.
Introduction: The Friedreich Ataxia Rating Scale-Activities of Daily Living (FARS-ADL) is a validated and highly utilized measure for evaluating patients with Friedreich Ataxia. While construct validity of FARS-ADL has been shown for spinocerebellar ataxia (SCA), content validity has not been established.
Methods: Individuals with SCA1 or SCA3 (n = 7) and healthcare professionals (HCPs) with SCA expertise (n = 8) participated in qualitative interviews evaluating the relevance, clarity, and clinical meaningfulness of FARS-ADL for assessment of individuals with SCA.
Patient experience with acute hepatic porphyria before and after long-term givosiran treatment in a qualitative interview study.
Mol Genet Metab Rep
March 2025
Alnylam Pharmaceuticals, Maidenhead, UK.
Background: Acute hepatic porphyria (AHP) is characterized by debilitating and potentially life-threatening neurovisceral attacks, possible chronic symptoms, and long-term complications. In a phase 1/2 open-label extension (OLE) study and the phase 3 ENVISION study, givosiran led to sustained improvement in annualized attack rate and quality of life (QOL) measures. To capture the patient experience of symptoms and impacts of AHP, and any changes experienced during treatment with givosiran, qualitative interviews were conducted with study participants.
View Article and Find Full Text PDFConducting clinical trials with self-collection of pharmacokinetic samples: Experience from an exploratory, phase 1, open-label trial of centanafadine SR in healthy individuals.
Contemp Clin Trials Commun
February 2025
Verily Life Sciences, 269 E Grand Ave., South San Francisco, CA, 94080, USA.
Background: The COVID-19 pandemic accelerated a shift to decentralized clinical trials. We present the potential feasibility of this approach from a phase 1 pharmacokinetic (PK) trial.
Methods: Healthy adults (18-55 years) with a body mass index of 19.
Methodological challenges using routine clinical care data for real-world evidence: a rapid review utilizing a systematic literature search and focus group discussion.
BMC Med Res Methodol
January 2025
Freiburg Center for Data Analysis, Modeling and AI, University of Freiburg, Freiburg, Germany.
Background: The integration of real-world evidence (RWE) from real-world data (RWD) in clinical research is crucial for bridging the gap between clinical trial results and real-world outcomes. Analyzing routinely collected data to generate clinical evidence faces methodological concerns like confounding and bias, similar to prospectively documented observational studies. This study focuses on additional limitations frequently reported in the literature, providing an overview of the challenges and biases inherent to analyzing routine clinical care data, including health claims data (hereafter: routine data).
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