Results 61 to 70 of about 36,350 (272)
Elevation of transaminases. What if not the liver?
Лечащий ВрачBackground. 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
A clear approach: Hemostatic gel as a novel adjunct for pediatric upper gastrointestinal bleeding
JPGN Reports, EarlyView.Abstract Pediatric upper gastrointestinal bleeding (UGIB) is a significant clinical concern, with a mortality rate of approximately 2%. Endoscopic management of UGIB in children includes various techniques such as injections, mechanical devices, thermal therapies, and topical agents.
Natalia Plott +5 more
wiley +1 more source
Frontiers in PediatricsThis case report describes initiation of Nocturnal Non-Invasive Ventilation in home settings for two adolescents with Duchenne Muscular Dystrophy and different neuropsychiatric and neurocognitive comorbidities: one has Autism Spectrum Disorder, and the ...
Pien M. M. Weerkamp +11 more
doaj +1 more source
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
Duchenne muscular dystrophy [PDF]
Thorax, 1999 In their recently published paper Simonds et al 1 importantly emphasised the desirability of using non-invasive intermittent positive pressure ventilation (IPPV) rather than tracheostomy for optimising quality of life in patients with hypercapnic Duchenne muscular dystrophy, and suggested that the use of nocturnal nasal IPPV can help to prolong ...
Y. ISHIKAWA, J. R BACH;, A K SIMONDS
openaire +2 more sources
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
Incorporating baseline covariates to validate surrogate endpoints with a constant biomarker under control arm [PDF]
arXiv, 2021 A surrogate endpoint S in a clinical trial is an outcome that may be measured earlier or more easily than the true outcome of interest T. In this work, we extend causal inference approaches to validate such a surrogate using potential outcomes. The causal association paradigm assesses the relationship of the treatment effect on the surrogate with the ...
arxiv
Muscle &Nerve, EarlyView.ABSTRACT Aims There are few long‐term studies evaluating clinical outcomes and mortality among individuals with Duchenne muscular dystrophy (DMD); particularly using longitudinal health administrative claims data, reflecting populations managed in typical clinical practice.
Christina Qian +6 more
wiley +1 more source