A Systematic Review of Approaches for Engaging Patients for Research on Rare Diseases

Patient engagement in research: a systematic review

(2014) Juan Pablo Domecq et al.   BMC HEALTH SERVICES RESEARCH

Public attitudes regarding a pilot study of newborn screening for spinal muscular atrophy

(2013) Erin Rothwell et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

The Art of Age-Appropriate Care

(2013) Lorna A. Fern et al.   CANCER NURSING

Methodology of clinical research in rare diseases: Development of a research program in juvenile neuronal ceroid lipofuscinosis (JNCL) via creation of a patient registry and collaboration with patient advocates

(2013) Elisabeth A. de Blieck et al.   Contemporary Clinical Trials

Patient And Family Engagement: A Framework For Understanding The Elements And Developing Interventions And Policies

(2013) Kristin L. Carman et al.   HEALTH AFFAIRS

Practical Aspects of Recruitment and Retention in Clinical Trials of Rare Genetic Diseases: The Phenylketonuria (PKU) Experience

(2013) Stephanie J. DeWard et al.   Journal of Genetic Counseling

Unresolved tensions in consumer engagement in CER: a US research perspective

(2013) Thomas Workman et al.   Journal of Comparative Effectiveness Research

Motivations of patients with pulmonary arterial hypertension to participate in randomized clinical trials

(2012) Ricki Carroll et al.   Clinical Trials

Research priorities of patients with neuromuscular disease

(2012) Christi J. Nierse et al.   DISABILITY AND REHABILITATION

How disease advocacy organizations participate in clinical research: a survey of genetic organizations

(2012) David C. Landy et al.   GENETICS IN MEDICINE

Cross-cultural adaptation of the CHO-KLAT for boys with hemophilia in rural and urban china

(2012) Runhui Wu et al.   Health and Quality of Life Outcomes

Continuous Patient Engagement in Comparative Effectiveness Research

(2012) Abdulla M. Abdulhalim   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

A New Taxonomy for Stakeholder Engagement in Patient-Centered Outcomes Research

(2012) Thomas W. Concannon et al.   JOURNAL OF GENERAL INTERNAL MEDICINE

The political empowerment of rare disease patient advocates both at EU and national level

(2012) Terkel Andersen   Orphanet Journal of Rare Diseases

Key outcomes from stakeholder workshops at a symposium to inform the development of an Australian national plan for rare diseases

(2012) Caron Molster et al.   Orphanet Journal of Rare Diseases

Li-Fraumeni syndrome: report of a clinical research workshop and creation of a research consortium

(2012) Phuong L. Mai et al.   Cancer Genetics

Looking back in time: conducting a cohort study of the long-term effects of treatment of adolescent tall girls with synthetic hormones

(2011) Fiona J Bruinsma et al.   BMC PUBLIC HEALTH

Consulting parents about the design of a randomized controlled trial of osteopathy for children with cerebral palsy

(2011) Vanessa Edwards et al.   HEALTH EXPECTATIONS

Establishing a consortium for the study of rare diseases: The Urea Cycle Disorders Consortium

(2010) Jennifer Seminara et al.   MOLECULAR GENETICS AND METABOLISM

The role of patient advocacy organisations in neuromuscular disease R&D – The case of the Dutch neuromuscular disease association VSN

(2010) Wouter Boon et al.   NEUROMUSCULAR DISORDERS

Establishment of a Community Advisory Board (CAB) for tuberculosis control and research in the Inanda, Ntuzuma and KwaMashu (INK) area of KwaZulu-Natal, South Africa

(2009) Sbongile P. Ntshanga et al.   HEALTH POLICY

Patient involvement in health research: A contribution to a systematic review on the effectiveness of treatments for degenerative ataxias

(2009) P. Serrano-Aguilar et al.   SOCIAL SCIENCE & MEDICINE

Patient-Important Outcomes in Registered Diabetes Trials

(2008) Gunjan Y. Gandhi   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION