The TREAT-NMD Duchenne muscular dystrophy registries: conception, design, and utilization by industry and academia. - Wikidata - awgldk - TriplyDB

The TREAT-NMD Duchenne muscular dystrophy registries: conception, design, and utilization by industry and academia.

The TREAT-NMD Duchenne muscular dystrophy registries: conception, design, and utilization by industry and academia.

http://www.wikidata.org/entity/Q38406108

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Current and emerging treatment strategies for Duchenne muscular dystrophy The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development 204th ENMC International Workshop on Biomarkers in Duchenne Muscular Dystrophy 24-26 January 2014, Naarden, The Netherlands The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review Early 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 research Nationwide patient registry for GNE myopathy in Japan The UK Myotonic Dystrophy Patient Registry: facilitating and accelerating clinical research The 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 Germany Adult 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 dystrophy Design, set-up and utility of the UK facioscapulohumeral muscular dystrophy patient registry The TREAT-NMD care and trial site registry: an online registry to facilitate clinical research for neuromuscular diseases Prednisolone improves walking in Japanese Duchenne muscular dystrophy patients Trends in steroid therapy for Duchenne muscular dystrophy in Japan European 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.

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Agnes Herczegfalvi

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Andriy V Shatillo

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Angela Stringer

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Angélica Moresco

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Anna Lusakowska

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Anna Roy

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Brigitta von Rekowski

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Can outcomes in Duchenne muscular dystrophy be improved by public reporting of data?

Complete cloning of the duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals

Advances in therapeutic RNA-targeting.

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations.

Copy number variation in the genome; the human DMD gene as an example.

Novel approaches to corticosteroid treatment in Duchenne muscular dystrophy

Characteristics of Japanese Duchenne and Becker muscular dystrophy patients in a novel Japanese national registry of muscular dystrophy (Remudy).

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management.

Corticosteroids in Duchenne muscular dystrophy: major variations in practice.

Characterization of the DMD/BMD patient population in Czech Republic and Slovakia using an innovative registry approach.

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Hanns Lochmüller

Janbernd Kirschner

Domenica Taruscio

Kevin M. Flanigan

Rosário Santos

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