Results 11 to 20 of about 54,565 (260)

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

Developing and Validating an Assessment Blueprint for Clinical Skill Competencies in General Surgery for the Medical Undergraduates [PDF]

Strides in Development of Medical Education, 2023
Background: Assessment is the requisite or essential part of medical education. A Blueprint is a calibrated or quality document that compiles the entire educational content and its outcome.Objectives: The present study was done to develop an assessment ...
Neetha Kundoor, Faiz Hussain, Sridhar Lingam, Venkat Narsimha Reddy, Rekha Arcot, Harshita Bondugula, Srilatha Bashetti   +6 more
doaj   +1 more source

A Blueprint for Involvement: Reflections of lived experience co-researchers and academic researchers on working collaboratively

Research Involvement and Engagement, 2022
Plain English summary The Blueprint study worked with young co-researchers with lived experience to explore services in England and Wales for children and young people with common mental health problems like depression, anxiety and self-harm.
The Blueprint Writing Collective
doaj   +1 more source

Partnering with patients to get better outcomes with chimeric antigen receptor T-cell therapy: towards engagement of patients in early phase trials

Research Involvement and Engagement, 2020
Aim Though patient engagement in clinical research is growing, recent reports suggest few clinical trials report on such activities. To address this gap, we describe our approach to patient engagement in the development of a clinical trial protocol to ...
Madison Foster, Dean A Fergusson, Terry Hawrysh, Justin Presseau, Natasha Kekre, Stuart Schwartz, Gisell Castillo, Sarah Asad, Grace Fox, Harold Atkins, Kednapa Thavorn, Joshua Montroy, Robert A Holt, Zarah Monfaredi, Manoj M Lalu   +14 more
doaj   +1 more source

Targeting kinases with precision

Molecular & Cellular Oncology, 2018
Cancer genomics and mechanistic studies have revealed that heterogeneous mutations within a single kinase can result in a variety of activation mechanisms.
Alexandra K. Gardino, Erica K. Evans, Joseph L. Kim, Natasja Brooijmans, Brian L. Hodous, Beni Wolf, Christoph Lengauer   +6 more
doaj   +1 more source

Opt‐in for secondary findings as part of diagnostic whole‐exome sequencing: Real‐life experience from an international diagnostic laboratory

Molecular Genetics & Genomic Medicine, 2023
Background Discussion about the risks and benefits of offering secondary findings as part of genome‐wide diagnostics lacks real‐life data. We studied the opt‐in decisions of patients/families referred to whole exome study (WES) in Blueprint Genetics (BpG)
Minna Brunfeldt, Milja Kaare, Inka Saarinen, Juha Koskenvuo, Helena Kääriäinen   +4 more
doaj   +1 more source

DSP p.(Thr2104Glnfs*12) variant presents variably with early onset severe arrhythmias and left ventricular cardiomyopathy

BMC Medical Genetics, 2020
Background Dilated cardiomyopathy (DCM) is a condition characterized by dilatation and systolic dysfunction of the left ventricle in the absence of severe coronary artery disease or abnormal loading conditions. Mutations in the titin (TTN) and lamin A/C (
Krista Heliö, Tiia Kangas-Kontio, Sini Weckström, Sari U. M. Vanninen, Katriina Aalto-Setälä, Tero-Pekka Alastalo, Samuel Myllykangas, Tiina M. Heliö, Juha W. Koskenvuo   +8 more
doaj   +1 more source

A Blueprint for Quality [PDF]

Canadian Journal of Gastroenterology, 2013
It is clear that enhancing the quality of endoscopic services improves patient outcomes. Benefits include shorter wait times, more appropriate screening and surveillance intervals, higher adenoma detection rates and fewer interval (or missed) cancers.
openaire   +4 more sources

Psychometric evaluation of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF©) and determination of a threshold score for moderate symptoms

Orphanet Journal of Rare Diseases, 2023
Background The Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF) (©Blueprint Medicines Corporation), a 12-item daily diary that assesses 11 signs and symptoms of indolent systemic mastocytosis (ISM) and smoldering systemic mastocytosis ...
Alan L. Shields, Fiona Taylor, Roger E. Lamoureux, Brad Padilla, Kas Severson, Tanya Green, Anthony L. Boral, Cem Akin, Frank Siebenhaar, Brenton Mar   +9 more
doaj   +1 more source

Single‐cell DNA methylation profiling: Technologies, computation, and applications in precision oncology

Molecular Oncology, EarlyView.
Single‐cell DNA methylation (scDNAme) profiling maps epimutational clonal evolution, revealing mechanisms of malignancy and therapeutic resistance across diverse cancer types. By providing a high‐resolution landscape of intratumoral heterogeneity, these technologies empower precise patient stratification, guide the development of enhanced ...
Ik Soo Kim
wiley   +1 more source