Results 51 to 60 of about 4,008,475 (239)

Initial Psychometric Evaluation of the Barth Syndrome Symptom Assessment (BTHS-SA) for Adolescents and Adults in a Phase 2 Clinical Study

Orphanet Journal of Rare Diseases
Background Barth syndrome (BTHS) is a rare, X-linked disorder that stems from mutations in the TAFAZZIN (TAZ) gene with varying disease severity among patients.
Chad Gwaltney, Alan Shields, Emily Love, Sarah Ollis, Jonathan Stokes, Iyar Mazar, Ethan Arenson, Anthony Aiudi, R. J. Wirth, Carrie Houts   +9 more
doaj   +1 more source

Deep Sequencing of FLT3‐ITD Enables Response Evaluation and Post‐Treatment Monitoring in Childhood AML: An Exploratory Study

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background An internal tandem duplication in the gene encoding Fms‐like tyrosine kinase 3 (FLT3‐ITD) is associated with high relapse risk and poor prognosis in acute myeloid leukemia (AML) and plays a crucial role in treatment decisions. Measurable residual disease (MRD) analysis of FLT3‐ITD during and after treatment has shown prognostic ...
Sofie Johansson Alm, Bea Tornberg, Erik Delsing Malmberg, Henrik Hasle, Kristian Løvvik Juul‐Dam, Lars Palmqvist, Borhan R. Saeed, Giti Shah Barkhordar, Anastasia Soboli, Anna Staffas, Jonas Abrahamsson, Linda Fogelstrand   +11 more
wiley   +1 more source

Development and content validity assessment of the Dry Eye Disease Questionnaire in patients with dry eye disease, meibomian gland dysfunction, and Sjögren’s syndrome dry eye disease

Journal of Patient-Reported Outcomes, 2023
Background Dry eye disease (DED), Meibomian gland dysfunction (MGD), and Sjögren’s syndrome dry eye disease (SS-DED) are eye dryness conditions that show significant overlap in various symptoms of ocular discomfort.
Brigitte Sloesen, Alyson Young, Katie Forde, Nicola Hodson, Sarah Bentley, Oonagh Walsh, Christel Naujoks, Paul O’Brien, Garima Sharma   +8 more
doaj   +1 more source

Claudin‐6 Protein Expression in Atypical Teratoid/Rhabdoid Tumors Is Strongly Enriched in the Molecular Subgroup AT/RT‐TYR

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Claudin‐6 has emerged as a promising immunotherapeutic target, yet protein‐level data in atypical teratoid/rhabdoid tumors (AT/RTs) have been inconsistent. We analyzed 36 well‐characterized AT/RT samples and found membranous claudin‐6 protein expression in 58% of cases, with striking enrichment in the molecular subgroup AT/RT‐TYR (100%) and ...
Victoria E. Fincke, Christian Thomas, Michael C. Frühwald, Pascal D. Johann, Martin Hasselblatt   +4 more
wiley   +1 more source

Patient Experience of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP): A Qualitative Exploration of Signs, Symptoms, and Health-Related Quality of Life Impacts

Neurology and Therapy
Introduction Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare type of autoimmune neuropathy, characterized by signs of distal and proximal weakness of the upper and lower limbs, sensory dysfunction, absent or diminished tendon reflexes,
Anna Roberts, Natasha Griffiths, Kieran Thiara, Sophie Wallace, Alyson L. Young, Nicola Williamson, Adam Gater, Omar Saeed, Charles Minor, Natalia Hawken   +9 more
doaj   +1 more source

Time Toxicity in Wilms Tumor: Quantifying the Burden of Healthcare Interaction in the First Year After Diagnosis

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Wilms tumor (WT) treatment imposes a significant time burden on patients and their families. Time toxicity is a patient‐centered metric that quantifies the burden of healthcare interaction. We sought to define time toxicity in the first year after diagnosis of WT and hypothesized that it would increase as tumor stage and treatment ...
Caleb Q. Ashbrook, Veerain Gupta, Nicholas J. Clark, Vijay Rings, Lauren E. Corona, Douglass B. Clayton, Daniel J. Benedetti   +6 more
wiley   +1 more source

Qualitative exploration of women’s experiences of vasomotor symptoms to support the content validity of patient-reported outcomes

Journal of Patient-Reported Outcomes
Background Frequency and severity of vasomotor symptoms (VMS; hot flashes) associated with menopause significantly impact women’s health-related quality of life (HRQoL). Treatment benefit in VMS clinical trials is assessed using patient-reported outcome (
Claudia Haberland, Melissa Barclay, Sophie Whyman, Asha Lehane, Adam Gater, Christoph Gerlinger, Christian Seitz, Maja Francuski, Nils Schoof, Andrew Trigg, Helena Bradley   +10 more
doaj   +1 more source

Psychometric evaluation of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF) in a phase 2 clinical study

Orphanet Journal of Rare Diseases, 2021
Background Indolent systemic mastocytosis (ISM) is a rare, clonal mast cell neoplasm characterized by severe, unpredictable symptoms. The Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF) items compose a Total Symptom Score (TSS ...
Brad Padilla, Alan L. Shields, Fiona Taylor, Xiaoran Li, Jeffrey Mcdonald, Tanya Green, Anthony L. Boral, Hui-Min Lin, Cem Akin, Frank Siebenhaar, Brenton Mar   +10 more
doaj   +1 more source

European Values Study 2017: Finnish Data

, 2019
Suomen European Values Study 2017 -aineisto on väestötutkimus, joka kartoittaa monipuolisesti suomalaisten moraalisia, uskonnollisia, yhteiskunnallisia ja sosiaalisia asenteita sekä arvoja.
European Values Study Group
core   +1 more source

Atlas of european values

, 2022
Do Europeans really feel European? Do they trust each other and are they solidary? What do they think of immigration and refugee influx? Do they want a greener and more sustainable Europe, and at what cost?

core   +1 more source