Results 51 to 60 of about 71,356 (167)

Automatic Classification of Neuromuscular Diseases in Children Using Photoacoustic Imaging [PDF]

arXiv, 2022
Neuromuscular diseases (NMDs) cause a significant burden for both healthcare systems and society. They can lead to severe progressive muscle weakness, muscle degeneration, contracture, deformity and progressive disability. The NMDs evaluated in this study often manifest in early childhood. As subtypes of disease, e.g.
arxiv  

Preimplantation genetic diagnosis associated to Duchenne muscular dystrophy

Einstein (São Paulo)
Duchenne muscular dystrophy is the most common muscle disease found in male children. Currently, there is no effective therapy available for Duchenne muscular dystrophy patients. Therefore, it is essential to make a prenatal diagnosis and provide genetic
Bianca Bianco, Denise Maria Christofolini, Gabriel Seixas Conceição, Caio Parente Barbosa   +3 more
doaj   +1 more source

Long-term Evaluation of AAV-CRISPR Genome Editing for Duchenne Muscular Dystrophy

Nature Network Boston, 2018
Duchenne muscular dystrophy (DMD) is a monogenic disorder and a candidate for therapeutic genome editing. There have been several recent reports of genome editing in preclinical models of Duchenne muscular dystrophy1–6, however, the long-term persistence
Christopher E. Nelson, Yaoying Wu, M. Gemberling, Matthew L. Oliver, Matthew A. Waller, Joel D Bohning, J. Robinson-Hamm, Karen Bulaklak, Ruth M. Castellanos Rivera, J. Collier, A. Asokan, C. Gersbach   +11 more
semanticscholar   +1 more source

Two-sample nonparametric test for proportional reversed hazards [PDF]

arXiv, 2022
Several works have been undertaken in the context of proportional reversed hazard rate (PRHR) since last few decades. But any specific statistical methodology for the PRHR hypothesis is absent in the literature. In this paper, a two-sample nonparametric test based on two independent samples is proposed for verifying the PRHR assumption.
arxiv  

Fat embolism after fractures in Duchenne muscular dystrophy: an underdiagnosed complication? A systematic review

Therapeutics and Clinical Risk Management, 2017
David Feder,1 Miriam Eva Koch,1 Beniamino Palmieri,2 Fernando Luiz Affonso Fonseca,1 Alzira Alves de Siqueira Carvalho3 1Pharmacology Department, Faculdade de Medicina do ABC, Santo André, São Paulo, Brazil; 2Department of General Surgery ...
Feder D, Koch ME, Palmieri B, Fonseca FLA, Carvalho AAS   +4 more
doaj  

Elevation of transaminases. What if not the liver?

Лечащий Врач
Background. According to Russian studies, the average age of Duchenne muscular dystrophy diagnosis is 7-8 years. This is because, on one hand, Duchenne muscular dystrophy is a rare disease, and a doctor may never see it throughout their clinical practice.
I. V. Sharkova
doaj   +1 more source

The Statistical Performance of Matching-Adjusted Indirect Comparisons [PDF]

arXiv, 2019
Indirect comparisons of treatment-specific outcomes across separate studies often inform decision-making in the absence of head-to-head randomized comparisons. Differences in baseline characteristics between study populations may introduce confounding bias in such comparisons. Matching-adjusted indirect comparison (MAIC) (Signorovitch et al., 2010) has
arxiv  

iASiS Open Data Graph: Automated Semantic Integration of Disease-Specific Knowledge [PDF]

arXiv, 2019
In biomedical research, unified access to up-to-date domain-specific knowledge is crucial, as such knowledge is continuously accumulated in scientific literature and structured resources. Identifying and extracting specific information is a challenging task and computational analysis of knowledge bases can be valuable in this direction.
arxiv  

ZIKQ: An innovative centile chart method for utilizing natural history data in rare disease clinical development [PDF]

Utilizing natural history data as external control plays an important role in the clinical development of rare diseases, since placebo groups in double-blind randomization trials may not be available due to ethical reasons and low disease prevalence. This article proposed an innovative approach for utilizing natural history data to support rare disease
arxiv   +1 more source

Automatic Segmentation of Muscle Tissue and Inter-muscular Fat in Thigh and Calf MRI Images [PDF]

, 2019
Magnetic resonance imaging (MRI) of thigh and calf muscles is one of the most effective techniques for estimating fat infiltration into muscular dystrophies. The infiltration of adipose tissue into the diseased muscle region varies in its severity across, and within, patients.
arxiv   +1 more source