Results 51 to 60 of about 273,377 (304)

Principles for interactions with biopharmaceutical companies: the development of guidelines for patient advocacy organizations in the field of rare diseases

Orphanet Journal of Rare Diseases, 2018
Background Rare diseases are a global public health concern, affecting an estimated 350 million individuals. Only 5% of approximately 7000 known rare diseases have a treatment, and only about half have a patient advocacy organization.
Susan Stein, Elizabeth Bogard, Nicole Boice, Vivian Fernandez, Tessa Field, Alan Gilstrap, Susan R. Kahn, Jane Larkindale, Toni Mathieson   +8 more
doaj   +1 more source

Vestibular Patient Journey: Insights From Vestibular Disorders Association (VeDA) Registry

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Vestibular symptoms impose a high burden of disability. Understanding real‐world diagnostic and treatment pathways can identify care gaps and guide interventions. We aimed to characterize symptom profiles, diagnostic trends, provider involvement, and treatment patterns in vestibular disorders.
Ali Rafati, Sami I. Nassar, Shaun A. Nguyen, Patricia L. Gerend, Michael C. Schubert, Michael T. Teixido, Frederick A. Godley, Joel A. Goebel, Cynthia A. Ryan, Habib G. Rizk, Amir Kheradmand   +10 more
wiley   +1 more source

An overview of the research evidence on ethnicity and communication in healthcare [PDF]

, 2005
• The aim of the present study was to identify and review the available research evidence on 'ethnicity and communication' in areas relevant to ensuring effective provision of mainstream services (e.g.
Clay, Diane, Gumber, Anil, HASH(0x55aa093742c0), Johnson, Mark R. D., Jones, Kip, Shaw, A., Szczepura, Ala   +6 more
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Caregivers' assessment of meaningful and relevant clinical outcome assessments for Sanfilippo syndrome

Journal of Patient-Reported Outcomes, 2022
Objectives Sanfilippo syndrome is a rare multisystem disease with no approved treatments. This study explores caregiver perspectives on the most impactful symptoms and patient-relevant clinical outcomes assessments.
Katherine Ackerman Porter, Cara O’Neill, Elise Drake, Sara M. Andrews, Kathleen Delaney, Samantha Parker, Maria L. Escolar, Stacey Montgomery, William Moon, Carolyn Worrall, Holly L. Peay   +10 more
doaj   +1 more source

RNA Sequencing Resolves Cryptic Pathogenic Variants in Mitochondrial Disease

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Mitochondrial diseases are the most common inherited metabolic disorders, characterized by pronounced clinical and genetic heterogeneity that complicates molecular diagnosis. Although DNA‐based sequencing approaches have become standard in genetic testing, up to half of patients remain without a definitive diagnosis.
Zhimei Liu, Xin Duan, Fatemeh Peymani, Jia Wang, Chengjia Bao, Chaolong Xu, Ying Zou, Zixuan Zhang, Yunxi Zhang, Tongyue Li, Martin Pavlov, Junling Wang, Minhan Song, Tianyu Song, Xiaodi Han, Mingxi Sun, Danmin Shen, Ruoyu Duan, Huafang Jiang, Manting Xu, Holger Prokisch, Fang Fang   +21 more
wiley   +1 more source

Considering Harm and Safety in Youth Mental Health: A Call for Attention and Action [PDF]

, 2014
The possibility of harm from mental health provision, and in particular harm from youth mental health provision, has been largely overlooked. We contend that if we continue to assume youth mental health services can do no harm, and all that is needed is ...
Deighton, J, Fleming, I, Lachman, P, Wolpert, M   +3 more
core   +1 more source

Factors Associated with Poor Mental Health in Patients with Axial Spondyloarthritis: Results from the International Map of Axial Spondyloarthritis (IMAS)

RMD Open
Background This study aims to assess the prevalence of poor mental health in axial spondyloarthritis (axSpA) and its associated factors in a large sample of patients from the International Map of Axial Spondyloarthritis (IMAS) study from around the globe.
Denis Poddubnyy, Victoria Navarro-Compán, Shashank Akerkar, Marco Garrido-Cumbrera, Souzi Makri, José Correa-Fernández, Christine Bundy, Fernando Sommerfleck, Jo Lowe, Elie Karam   +9 more
doaj   +1 more source

2012 Annual Report: Achieving Access to Health for All Coloradans [PDF]

, 2013
This annual report includes: an 'about us' introduction, a letter from the leadership, details of grantmaking activities in the areas of health data and information, health advocacy and policy, health care services and systems, and health and well-being,

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A Qualitative Analysis of Patient Perspectives and Preferences in Lupus Management to Guide Lupus Guidelines Development

Arthritis Care &Research, EarlyView.
Objective A patient‐centered approach for chronic disease management, including systemic lupus erythematosus (SLE), aligns treatment with patients’ values and preferences, leading to improved outcomes. This paper summarizes how patient experiences, perspectives, and priorities informed the American College of Rheumatology (ACR) 2024 Lupus Nephritis (LN)
Shivani Garg, Izzy Hartel, Lisa R. Sammaritano, Anca Askanase, Bonnie L. Bermas, Maria Dall'Era, Alí Duarte‐García, Victoria P. Werth, Brad Rovin, Reem A. Mustafa, Amy S. Turner, Bené Williams, Brian L. Ung, Hiya Bhavsar, Monique C. Gore‐Massy, Nahirannette Pulido, Natacha Guerrero, Natalie M. Smith, Wambui Machua, Linda T. Hiraki, Mary Beth Son   +20 more
wiley   +1 more source

Advocacy Coalition Framework Lens on Pressing Healthcare Issues

, 2020
In deciding how to interpret and understand public policy, many experts use theories and frameworks to justify their reasoning. One of the most common avenue of viewing policy involves the advocacy coalition framework based on its broad applicability ...
Goodwin, Garrett S.
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