Publications by authors named "Allison McCrady"

Introduction/aims: Improved methodologies to monitor the progression of Duchenne muscular dystrophy (DMD) are needed, especially in the context of clinical trials. We report changes in muscle magnetic resonance imaging (MRI) parameters in participants with DMD, including changes in lean muscle volume (LMV), muscle fat fraction (MFF), and muscle fat infiltration (MFI) and their relationship to changes in functional parameters.

Methods: MRI data were obtained as part of a clinical study (NCT02310763) of domagrozumab, an antibody-targeting myostatin that negatively regulates skeletal muscle mass.

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Introduction/aims: Spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD) are progressive neuromuscular disorders characterized by severe muscle weakness and functional decline (Pillen et al., Muscle Nerve 2008; 37(6):679-693). With new therapeutics, objective methods with increased sensitivity are needed to assess muscle function.

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Background: Neuromuscular diseases, such as spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD), may result in the loss of motor movements, respiratory failure, and early mortality in young children and in adulthood. With novel treatments now available, new evaluation methods are needed to assess progress that is not currently captured in existing motor scale tests.

Objective: With our feasibility study, our interdisciplinary team of investigators aims to develop a novel, multimodal paradigm of measuring motor function in children with neuromuscular diseases that will revolutionize the way that clinical trial end points are measured, thereby accelerating the pipeline of new treatments for childhood neuromuscular diseases.

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