Search Results - "Malkus, Elizabeth C"
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One Year Outcome of Boys With Duchenne Muscular Dystrophy Using the Bayley-III Scales of Infant and Toddler Development
Published in Pediatric neurology (01-06-2014)“…Abstract Background The pathogenesis of Duchenne muscular dystrophy starts before birth. Despite this, clinical trials exclude young boys because traditional…”
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Muscle ultrasound quantifies disease progression over time in infants and young boys with duchenne muscular dystrophy
Published in Muscle & nerve (01-09-2015)“…ABSTRACT Introduction: Quantitative muscle ultrasound (QUS) in boys with Duchenne muscular dystrophy (DMD) shows increased echointensity as muscle is replaced…”
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Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy
Published in Muscle & nerve (01-04-2015)“…ABSTRACT Introduction: Therapeutic trials in Duchenne muscular dystrophy (DMD) often exclude non‐ambulatory individuals. Here we establish optimal and reliable…”
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Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network
Published in Neuromuscular disorders : NMD (01-07-2013)“…Abstract Therapeutic trials in Duchenne Muscular Dystrophy (DMD) exclude young boys because traditional outcome measures rely on cooperation. The Bayley III…”
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Quantitative ultrasound using backscatter analysis in Duchenne and Becker muscular dystrophy
Published in Neuromuscular disorders : NMD (01-12-2010)“…Abstract Evaluation of ultrasound images of muscle with calibrated muscle backscatter (cMB) provides reproducible quantitative measurements of muscle…”
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Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up
Published in Muscle & nerve (01-10-2016)“…ABSTRACT Introduction: Outcomes sensitive to change over time in non‐ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well‐established…”
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Qualitative and quantitative skeletal muscle ultrasound in late-onset acid maltase deficiency
Published in Muscle & nerve (01-09-2011)“…Introduction: Acid maltase deficiency (AMD, or Pompe disease) is an inherited myopathic disorder of glycogen degradation. Diagnosis is often delayed. Muscle…”
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Characterization of Strength and Function in Ambulatory Adults With GNE Myopathy
Published in Journal of clinical neuromuscular disease (01-09-2017)“…OBJECTIVE:To characterize the pattern and extent of muscle weakness and impact on physical functioning in adults with GNEM. METHODS:Strength and function were…”
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Motor Function Test Reliability During the NeuroNEXT Spinal Muscular Atrophy Infant Biomarker Study
Published in Journal of neuromuscular diseases (2018)“…The NeuroNEXT SMA Infant Biomarker Study, a two year, longitudinal, multi-center study of infants with SMA type 1 and healthy infants, presented a unique…”
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A multinational study on motor function in early-onset FSHD
Published in Neurology (10-04-2018)“…OBJECTIVESTo investigate motor function associations with age, sex, and D4Z4 repeats among participants with early-onset facioscapulohumeral muscular dystrophy…”
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