Results 71 to 80 of about 36,350 (272)

Fat embolism syndrome in a child with muscular dystrophy of Duchenne type bilateral femur fracture. A rare association.

open access: yesRevista de la Facultad de Medicina, 2007
It is high the frequency of femur fracture in children with Duchenne Muscular Dystrophy (DMD), with an incidence between 15 and 44%. The fatty embolism is presented in fractures of long bones generally associated to trauma of high energy or to extensive ...
Enrique Vergara Amador   +2 more
doaj  

Duchenne Muscular Dystrophy Newborn Screening, a Case Study for Examining Ethical and Legal Issues for Pilots for Emerging Disorders: Considerations and Recommendations

open access: yesInternational Journal of Neonatal Screening, 2018
Duchenne muscular dystrophy (DMD/Duchenne) is one of the ten most severe and common pediatric genetic diseases and affects an estimated 1 in every 5000 male births.
Michele A. Lloyd-Puryear   +9 more
doaj   +1 more source

Detecting Multiple Replicating Signals using Adaptive Filtering Procedures [PDF]

open access: yesarXiv, 2016
Replicability is a fundamental quality of scientific discoveries: we are interested in those signals that are detectable in different laboratories, study populations, across time etc. Unlike meta-analysis which accounts for experimental variability but does not guarantee replicability, testing a partial conjunction (PC) null aims specifically to ...
arxiv  

Myostatin Knockout Mice Have Larger Muscle Fibers With Normal Function and Morphology

open access: yesMuscle &Nerve, EarlyView.
ABSTRACT Introduction We assessed whether muscle fibers in myostatin knockout (MSTN−/−) mice are just larger or also exhibit morphological, metabolic, and functional differences from MSTN+/+ mice. Methods We compared single fiber contractile properties and histological fiber properties in muscles from MSTN−/− and MSTN+/+ mice.
Hans Degens, Ketan Patel, A. Matsakas
wiley   +1 more source

Non-operative treatment for perforated gastro-duodenal peptic ulcer in Duchenne Muscular Dystrophy: a case report

open access: yesBMC Surgery, 2004
Background Clinical characteristics and complications of Duchenne muscular dystrophy caused by skeletal and cardiac muscle degeneration are well known. Gastro-intestinal involvement has also been recognised in these patients.
Wever Jan   +4 more
doaj  

Correlation of Utrophin Levels with the Dystrophin Protein Complex and Muscle Fibre Regeneration in Duchenne and Becker Muscular Dystrophy Muscle Biopsies.

open access: yesPLoS ONE, 2016
Duchenne muscular dystrophy is a severe and currently incurable progressive neuromuscular condition, caused by mutations in the DMD gene that result in the inability to produce dystrophin. Lack of dystrophin leads to loss of muscle fibres and a reduction
Narinder Janghra   +6 more
doaj   +1 more source

Challenges in the management of the child with Duchenne muscular dystrophy in a resource poor setting:a case report

open access: yesThe Pan African Medical Journal, 2014
Duchenne muscular dystrophy is a progressive genetic disease with no cure at present. Children suffering from this disease eventually become wheelchair bound and die in their late teens.
Kelechi Kenneth Odinaka   +1 more
doaj   +1 more source

Outcome of Long-Term Corticosteroid Treatment in Duchenne Muscular Dystrophy

open access: yesPediatric Neurology Briefs, 2007
The clinical orthopedic effects of chronic daily corticosteroid treatment were evaluated by chart review in boys with genetically confirmed Duchenne muscular dystrophy (DMD) followed at the Ohio State University Muscular Dystrophy Clinic between 2000 and
J Gordon Millichap
doaj   +1 more source

A model‐informed clinical trial simulation tool with a graphical user interface for Duchenne muscular dystrophy

open access: yesCPT: Pharmacometrics &Systems Pharmacology, EarlyView.
Abstract Quantitative model‐based clinical trial simulation tools play a critical role in informing study designs through simulation before actual execution. These tools help drug developers explore various trial scenarios in silico to select a clinical trial design to detect therapeutic effects more efficiently, therefore reducing time, expense, and ...
Jongjin Kim   +10 more
wiley   +1 more source

A computational tool to optimize clinical trial parameter selection in Duchenne muscular dystrophy: A practical guide and case studies

open access: yesCPT: Pharmacometrics &Systems Pharmacology, EarlyView.
Abstract Duchenne muscular dystrophy (DMD), a rare pediatric disease, presents numerous challenges when designing clinical trials, mainly due to the scarcity of available trial participants and the heterogeneity of disease progression. A quantitative clinical trial simulator (CTS) has been developed based on previously published five disease ...
Jordan Wilk   +18 more
wiley   +1 more source

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