Natural history study duchenne

Prednisone/prednisolone and ... - PubMed Central (PMC)

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9/22/2015 · The Cooperative International Neuromuscular Research Group Duchenne Natural History Study: glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures. Muscle Nerve 2013; 48:55–67.

Prednisone/prednisolone and ... - PubMed Central (PMC)

CINRG Duchenne Natural History - cinrgresearch.org

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Duchenne Natural History. The Cooperative International Neuromuscular Research Group (CINRG) has established the largest prospective multicenter natural history study to date in Duchenne muscular dystrophy (DMD), the CINRG Duchenne Natural History Study (DNHS). Since 2006 the study enrolled over 400 DMD boys and young men.

CINRG Duchenne Natural History - cinrgresearch.org

CINRG Duchenne Natural History Study demonstrates ...

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Through the Cooperative International Neuromuscular Research Group (CINRG), a clinical research network of academic centers, we collected natural history data from all participants in the CINRG Duchenne Natural History Study (DNHS) enrolled from 2006 to 2009. 8,9 These data provide the basis for a comprehensive description of cardiac disease in ...

CINRG Duchenne Natural History Study demonstrates ...

A Natural History Study In Chinese Male Patients With ...

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A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government.

A Natural History Study In Chinese Male Patients With ...

A Prospective Natural History Study of Progression of ...

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A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy. The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government.

A Prospective Natural History Study of Progression of ...

Natural History Studies in Duchenne Muscular Dystrophy ...

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CINRG Natural History Study and Development of Clinical Endpoints in Duchenne Muscular Dystrophy Craig McDonald, MD www.cinrgresearch.org CINRG Clinical Sites CINRG Clinical Site Locations US Sites International Sites University Hospitals, Leuven, Belgium Hadassah, Hebrew University Hospital,

Natural History Studies in Duchenne Muscular Dystrophy ...

CINRG Duchenne Natural History Studies - Parent Project ...

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The CINRG Duchenne Natural History Study (DNHS) is the most comprehensive study of Duchenne clinical outcomes and disease progression to date. Over 400 boys and men across the world with Duchenne participated in the study, making it the largest Duchenne natural history study ever.

CINRG Duchenne Natural History Studies - Parent Project ...

NMD Natural History Studies - trinds.com

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This study is the largest and longest Duchenne natural history study to date and is providing critical information to the Duchenne community. The study enrolled over 400 Duchenne boys and young men and followed their disease course for over 5 years and in some cases, close to 10 years.

NMD Natural History Studies - trinds.com

The natural history of the patients with Duchenne muscular ...

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The natural history of the patients with Duchenne muscular dystrophy in Taiwan: A medical center experience ... This retrospective study analyzed the natural history of patients with DMD and evaluated the effect of steroid therapy, which has never been documented before in Taiwan. ... Genotypes of the patients with Duchenne muscular dystrophy.

The natural history of the patients with Duchenne muscular ...

Becker Natural History Study - Parent Project Muscular ...

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What is the goal or purpose of this study? This is a natural history study to characterize the Becker muscular dystrophy clinical presentation and also to provide information regarding the possible effectiveness of a therapy currently in development for Duchenne muscular dystrophy (exon skipping).

Becker Natural History Study - Parent Project Muscular ...

Natural History Studies and Measuring Effectiveness of ...

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Natural History Studies and Measuring Effectiveness of Care in Duchenne Muscular Dystrophy Craig M. McDonald, MD Professor and Chair Physical Medicine & Rehabilitation Professor of Pediatrics University of California Davis School of Medicine

Natural History Studies and Measuring Effectiveness of ...

Genetic modifiers of ambulation in the cooperative ...

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The Cooperative International Neuromuscular Research Group Duchenne Natural History Study: glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures. Muscle Nerve 2013; 48: 55 – 67.

Genetic modifiers of ambulation in the cooperative ...

A study on natural history of Duchenne muscular dystrophy ...

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Objective: To study natural history and genotype of DMD in selected cohort of DMD patients. Background: Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy affecting one in 3,500 live male births. There are no studies from India to describe natural history of DMD and role of geo-ethnic factors on natural history of motor milestone in DMD compared to Caucasian patients.

A study on natural history of Duchenne muscular dystrophy ...

The cooperative international neuromuscular research group ...

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Next article in issue: The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures

The cooperative international neuromuscular research group ...

Longitudinal pulmonary function testing outcome measures ...

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We describe changes in pulmonary function measures across time in Duchenne muscular dystrophy patients treated with glucocorticoids (GCs) > 1 year compared to GC naïve patients in the Cooperative International Research Group Duchenne Natural History Study, a multicenter prospective cohort study. 397 participants underwent 2799 pulmonary function assessments over a period up to 10 years.

Longitudinal pulmonary function testing outcome measures ...

DMD genotypes and loss of ambulation in the CINRG Duchenne ...

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PDF | Objective: To correlate time to loss of ambulation (LoA) and different truncating DMD gene mutations in a large, prospective natural history study of Duchenne muscular dystrophy (DMD), with ...

DMD genotypes and loss of ambulation in the CINRG Duchenne ...

The Cooperative International Neuromuscular Research Group ...

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The Cooperative International Neuromuscular Research Group Duchenne Natural History Study-A Longitudinal Investigation In The Era Of Glucocorticoid Therapy: Design …

The Cooperative International Neuromuscular Research Group ...

Prednisone/prednisolone and deflazacort ... - Neurology

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9/22/2015 · Objective: We aimed to perform an observational study of age at loss of independent ambulation (LoA) and side-effect profiles associated with different glucocorticoid corticosteroid (GC) regimens in Duchenne muscular dystrophy (DMD). Methods: We studied 340 participants in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG …

Prednisone/prednisolone and deflazacort ... - Neurology

Prosensa Completes Enrollment in Natural History Study of ...

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Prosensa Completes Enrollment in Natural History Study of Duchenne Muscular Dystrophy . Leiden, The Netherlands, June 25, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced the successful completion of enrollment in its prospective Natural …

Prosensa Completes Enrollment in Natural History Study of ...

Discovery of Metabolic Biomarkers for Duchenne Muscular ...

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Serum metabolite profiling in Duchenne muscular dystrophy (DMD) may enable discovery of valuable molecular markers for disease progression and treatment response. Serum samples from 51 DMD patients from a natural history study and 22 age-matched healthy volunteers were profiled using liquid chromatography coupled to mass spectrometry (LC-MS) for discovery of novel circulating serum …

Discovery of Metabolic Biomarkers for Duchenne Muscular ...

TRiNDS

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CINRG has established the largest and longest Duchenne natural history study to date. It has become an invaluable resource to the Duchenne and rare disease community at large. It has helped identify improvements in standards of care as well as possible gaps, development of clinical trial endpoints and composite measures, as well as sources of ...

TRiNDS
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