Results 61 to 70 of about 71,888 (256)

Global Equity in Clinical Trials: A Pragmatic Approach

JCO Global Oncology
Hussain I. Rangoonwala, Jennifer S. Morgan, Elias Melly, Abraham Siika, Patrick J. Loehrer, Naftali Busakhala   +5 more
doaj   +3 more sources

Pragmatic clinical trials in the context of regulation of medicines [PDF]

Upsala Journal of Medical Sciences, 2018
The pragmatic clinical trial addresses scientific questions in a setting close to routine clinical practice and sometimes using routinely collected data. From a regulatory perspective, when evaluating a new medicine before approving marketing authorization, there will never be enough patients studied in all subgroups that may potentially be at higher ...
Gedeborg, Rolf, Cline, Charles, Zethelius, Björn, Salmonson, Tomas   +3 more
openaire   +4 more sources

Upper Cervical Cord Area as a Biomarker of Conversion to Secondary Progressive Multiple Sclerosis

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective This study assessed whether upper cervical cord area (UCCA) measured on routine brain MRI can serve as a biomarker of conversion to SPMS. Methods This is a single‐center retrospective cohort study of RRMS patients with cross‐sectional and longitudinal analyses of clinical and MRI data. Future SPMS converters were matched by age, sex,
Nabil K. El Ayoubi, Sola Al Ghazal, Salem Hannoun, Medhat A. Siddik, Nour M. Najjar, Jim Dagher, Joseph Abboud, Myriam G. Semaan, Samia J. Khoury   +8 more
wiley   +1 more source

The ethical justification for inclusion of neonates in pragmatic randomized clinical trials for emergency newborn care

BMC Pediatrics, 2019
Background Research guidelines generally recognize vulnerable populations to include neonates with the aim of enhancing protections from harm. In practice, such guidance results in limiting participation in randomized clinical trials (RCTs).
Dan Kabonge Kaye
doaj   +1 more source

The Multiple Sclerosis Severity Allele rs10191329A and Cognitive Function: A UK Biobank Study

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT The genome‐wide association study of Multiple Sclerosis severity linked the genetic variant rs10191329A to long‐term disability and implicated brain resilience as a determinant of outcome. We hypothesised that rs10191329A might influence cognition in other neurological diseases and healthy controls.
Ioanna Zimianiti, Sheena Waters, Adil Harroud, Pernilla Stridh, Ruth Dobson, Benjamin M. Jacobs   +5 more
wiley   +1 more source

Positive predictive value of respiratory infection diagnoses in the Danish National Patient Registry: A post hoc validation analysis of the DANFLU-1 trial

International Journal of Infectious Diseases
Objectives: To validate respiratory infection diagnosis codes in the Danish National Patient Registry (DNPR) in the DANFLU-1 feasibility trial, and to assess whether reperforming effectiveness analyses with validated events altered findings.
Jacob Christensen, Niklas Dyrby Johansen, Katrine Feldballe Bernholm, Julie Inge-Marie Helene Borchsenius, Kira Hyldekær Janstrup, Daniel Modin, Kristoffer Grundtvig Skaarup, Joshua Nealon, Sandrine Samson, Matthew Loiacono, Youjun Huang, Rebecca Harris, Carsten Schade Larsen, Anne Marie Reimer Jensen, Nino Emanuel Landler, Brian L. Claggett, Scott D. Solomon, Gunnar H. Gislason, Lars Køber, Martin J. Landray, Pradeesh Sivapalan, Jens Ulrik Stæhr Jensen, Tor Biering-Sørensen   +22 more
doaj   +1 more source

Advancing Stroke Clinical Trials Using Community Engagement and Implementation Science Approaches

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Stroke clinical trials are essential for advancing stroke care but can face challenges with recruitment, retention, clinical relevance, and translation into real‐world practice. We propose that integrating community engagement and implementation science approaches into stroke trials can help address these needs.
Lesli E. Skolarus, Devin L. Brown, Bernadette Boden‐Albala   +2 more
wiley   +1 more source

Deep Learning Pose Estimation for Phenotyping of Co‐Occurring Hyperkinetic Movement Disorders

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective To explore whether routine outpatient video combined with deep learning‐based pose estimation and clinically interpretable kinematic features can support multi‐label phenotyping of co‐occurring hyperkinetic movement disorders (HMDs).
Laura Cif, Diane Demailly, Gabriella A. Horvàth, Juan Dario Ortigoza Escobar, Nathalie Dorison, Mayté Castro Jiménez, Cécile A. Hubsch, Thomas Wirth, Gun‐Marie Hariz, Sophie Huby, Morgan Dornadic, Zohra Souei, Muhammad Mushhood Ur Rehman, Simone Hemm, Mehdi Boulaymen, Eduardo M. Moraud, Jocelyne Bloch, Xavier Vasques   +17 more
wiley   +1 more source

Re‐Purposing Sapropterin (Kuvan) for ACTA2‐Related Multisystemic Smooth Muscle Dysfunction Syndrome: A Translational Mechanistic and First‐In‐Human Therapeutic Report

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Multisystemic smooth muscle dysfunction syndrome (MSMDS) is an ultra‐rare, ACTA2‐related disorder characterized by severe cerebrovascular disease, aortic aneurysms, and smooth muscle dysfunction. Using molecular dynamics simulations and in silico drug screening, we identified that sapropterin dihydrochloride (Kuvan) is a candidate capable of ...
Moran Hausman‐Kedem, Noa Bielopolski, Vijai Krishnan, Shane Wald‐Altman, Shelly I Shrian, Omer Bar‐Yosef, Livia Kapusta, Claire Shamber, Patricia L. Musolino, Miguel Weil   +9 more
wiley   +1 more source

Miglustat as a Treatment for Adults with Tangier Disease Neuropathy: The MUSTANG N-of-1 Trial with 21 months Clinical Observation

Neurology and Therapy
Importance Tangier disease (TD) is an ultra-rare disease, characterised by progressive peripheral neuropathy with no established treatment. Objectives To determine whether miglustat improved the clinical status of a single patient with TD, and to ...
Andrew Cook, Beth Stuart, Antonio Ochoa-Ferraro, Nicola Condon, Megan Lawrence, Fran Webley, Kerri-Lee Wallom, Claire Forbes, Vishy Veeranna, Subadra Wanninayake, Tom Oliver, Nicholas Davies, Charlotte Dawson, Frances Platt, Tarekegn Geberhiwot   +14 more
doaj   +1 more source