The TREAT-NMD Duchenne muscular dystrophy registries: conception, design, and utilization by industry and academia.
about
Current and emerging treatment strategies for Duchenne muscular dystrophyThe TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development204th ENMC International Workshop on Biomarkers in Duchenne Muscular Dystrophy 24-26 January 2014, Naarden, The NetherlandsThe burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence reviewEarly stages of building a rare disease registry, methods and 2010 data from the Belgian Neuromuscular Disease Registry (BNMDR).Patient/family views on data sharing in rare diseases: study in the European LeukoTreat project.'You should at least ask'. The expectations, hopes and fears of rare disease patients on large-scale data and biomaterial sharing for genomics researchNationwide patient registry for GNE myopathy in JapanThe UK Myotonic Dystrophy Patient Registry: facilitating and accelerating clinical researchThe burden of Duchenne muscular dystrophy: an international, cross-sectional study.TOSCA - first international registry to address knowledge gaps in the natural history and management of tuberous sclerosis complex.Comparative cost of illness analysis and assessment of health care burden of Duchenne and Becker muscular dystrophies in GermanyAdult care for Duchenne muscular dystrophy in the UK.Comparison of mutation profiles in the Duchenne muscular dystrophy gene among populations: implications for potential molecular therapies.The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations.A 26-Year Experience in Chorionic Villus Sampling Prenatal Genetic Diagnosis.Clinical trial network for the promotion of clinical research for rare diseases in Japan: muscular dystrophy clinical trial network.Quantifying the burden of caregiving in Duchenne muscular dystrophyDesign, set-up and utility of the UK facioscapulohumeral muscular dystrophy patient registryThe TREAT-NMD care and trial site registry: an online registry to facilitate clinical research for neuromuscular diseasesPrednisolone improves walking in Japanese Duchenne muscular dystrophy patientsTrends in steroid therapy for Duchenne muscular dystrophy in JapanEuropean Cross-Sectional Survey of Current Care Practices for Duchenne Muscular Dystrophy Reveals Regional and Age-Dependent Differences.Looking Forward to New Therapies: A Personal Perspective on the Translational Landscape for Muscular Dystrophies.'IRDiRC Recognized Resources': a new mechanism to support scientists to conduct efficient, high-quality research for rare diseases.International Cooperation to Enable the Diagnosis of All Rare Genetic Diseases.A fine balance and a shared learning journey: Exploring healthcare engagement through the experiences of youth with Neuromuscular Disorders.Comprehensive analysis for genetic diagnosis of Dystrophinopathies in Japan.The New Zealand Neuromuscular Disease Patient Registry; Five Years and a Thousand Patients.Predictors of Health-Related Quality of Life in boys with Duchenne muscular dystrophy from six European countries.Social/economic costs and health-related quality of life in patients with Duchenne muscular dystrophy in Europe.Factors Associated With Health-Related Quality of Life in Children With Duchenne Muscular Dystrophy.Towards a European Registry and Biorepository for Patients with Spinal and Bulbar Muscular Atrophy.Recent developments in Duchenne muscular dystrophy: facts and numbers.Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database.Natural History, Trial Readiness and Gene Discovery: Advances in Patient Registries for Neuromuscular Disease.Linked Registries: Connecting Rare Diseases Patient Registries through a Semantic Web Layer.215th ENMC International Workshop VCP-related multi-system proteinopathy (IBMPFD) 13-15 November 2015, Heemskerk, The Netherlands.Phenotypic stratification and genotype-phenotype correlation in a heterogeneous, international cohort of GNE myopathy patients: First report from the GNE myopathy Disease Monitoring Program, registry portion.Psychometric properties of the Zarit Caregiver Burden Interview administered to caregivers to patients with Duchenne muscular dystrophy: a Rasch analysis.
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The TREAT-NMD Duchenne muscular dystrophy registries: conception, design, and utilization by industry and academia.
description
2013 nî lūn-bûn
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2013年の論文
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2013年論文
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2013年論文
@zh-hant
2013年論文
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2013年論文
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2013年論文
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2013年论文
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2013年论文
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2013年论文
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name
The TREAT-NMD Duchenne muscula ...... tion by industry and academia.
@en
type
label
The TREAT-NMD Duchenne muscula ...... tion by industry and academia.
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prefLabel
The TREAT-NMD Duchenne muscula ...... tion by industry and academia.
@en
P2093
P2860
P50
P356
P1433
P1476
The TREAT-NMD Duchenne muscula ...... tion by industry and academia.
@en
P2093
Agnes Herczegfalvi
Andriy V Shatillo
Angela Stringer
Angélica Moresco
Anna Ambrosini
Anna Lusakowska
Ayşe Karaduman
Björn Lindvall
Brigitta von Rekowski
P2860
P304
P356
10.1002/HUMU.22390
P50
P577
2013-08-26T00:00:00Z