A journey of hope: lessons learned from studies on rare diseases and orphan drugs.
about
The quality of economic evaluations of ultra-orphan drugs in Europe - a systematic reviewScoping review of patient- and family-oriented outcomes and measures for chronic pediatric diseaseAn innovative and collaborative partnership between patients with rare disease and industry-supported registries: the Global aHUS RegistryThe National Clinical Trials Network: Conducting Successful Clinical Trials of New Therapies for Rare CancersMulti-criteria decision analysis (MCDA): testing a proposed MCDA framework for orphan drugsA methodology for a minimum data set for rare diseases to support national centers of excellence for healthcare and research.Lessons learnt from assembling screening libraries for drug discovery for neglected diseases.Access to orphan drugs despite poor quality of clinical evidence.The need for worldwide policy and action plans for rare diseases.Unintended effects of orphan product designation for rare neurological diseases.The involvement of patient organisations in rare disease research: a mixed methods study in Australia.Challenges in the design and conduct of therapeutic trials in channel disorders.New approaches to the treatment of dense deposit disease.Directions for new developments on statistical design and analysis of small population group trialsMethodology 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.Incentives for orphan drug research and development in the United StatesPredictors of orphan drug approval in the European Union.What, if anything, is specific about having a rare disorder? Patients' judgements on being ill and being rare.The RUDY study platform - a novel approach to patient driven research in rare musculoskeletal diseasesEthical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients' health needs?New directions for patient-centred care in scleroderma: the Scleroderma Patient-centred Intervention Network (SPIN).Emergence of pediatric rare diseases: Review of present policies and opportunities for improvementReview of 11 national policies for rare diseases in the context of key patient needs.Ethical perspectives on relations between industry and neuropsychiatric medicine.Drug repositioning for orphan diseases.Involving patients in reducing decision uncertainties around orphan and ultra-orphan drugs: a rare opportunity?Cognitive and Affective Uses of a Thoracic Outlet Syndrome Facebook Support Group.Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe?Living with and treating rare diseases: experiences of patients and professional health care providers.Obtaining a genetic diagnosis in a child with disability: impact on parental quality of life.Opportunities for developing therapies for rare genetic diseases: focus on gain-of-function and allostery.How disease advocacy organizations participate in clinical research: a survey of genetic organizations.Living with a rare disorder: a systematic review of the qualitative literature.Orphan diseases: state of the drug discovery art.A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study.Linked Registries: Connecting Rare Diseases Patient Registries through a Semantic Web Layer.Three-dimensional cell culture: A powerful tool in tumor research and drug discovery.RenalTube: a network tool for clinical and genetic diagnosis of primary tubulopathies.A cross-national comparative study of orphan drug policies in the United States, the European Union, and Japan: towards a made-in-China orphan drug policy.Using a meta-narrative literature review and focus groups with key stakeholders to identify perceived challenges and solutions for generating robust evidence on the effectiveness of treatments for rare diseases.
P2860
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P2860
A journey of hope: lessons learned from studies on rare diseases and orphan drugs.
description
2006 nî lūn-bûn
@nan
2006年の論文
@ja
2006年論文
@yue
2006年論文
@zh-hant
2006年論文
@zh-hk
2006年論文
@zh-mo
2006年論文
@zh-tw
2006年论文
@wuu
2006年论文
@zh
2006年论文
@zh-cn
name
A journey of hope: lessons learned from studies on rare diseases and orphan drugs.
@ast
A journey of hope: lessons learned from studies on rare diseases and orphan drugs.
@en
type
label
A journey of hope: lessons learned from studies on rare diseases and orphan drugs.
@ast
A journey of hope: lessons learned from studies on rare diseases and orphan drugs.
@en
prefLabel
A journey of hope: lessons learned from studies on rare diseases and orphan drugs.
@ast
A journey of hope: lessons learned from studies on rare diseases and orphan drugs.
@en
P2860
P1476
A journey of hope: lessons learned from studies on rare diseases and orphan drugs.
@en
P2093
P2860
P356
10.1111/J.1365-2796.2006.01666.X
P407
P50
P577
2006-07-01T00:00:00Z