Publications by authors named "M Dimachkie"
Article Synopsis
- - The PROPEL trial studied the effects of cipaglucosidase alfa plus miglustat (cipa+mig) versus alglucosidase alfa plus placebo (alg+pbo) on adults with late-onset Pompe disease (LOPD) over 52 weeks, finding improvements in motor and respiratory function for those switching to cipa+mig.
- - Patient-reported outcomes (PROs) evaluated included various measures of physical function, fatigue, and overall quality of life, with statistical analyses comparing responses between the two treatment groups.
- - Results showed that cipa+mig significantly improved patient-reported impressions of change in ability to move around and generally outperformed alg+pbo in most PRO measures, indicating enhanced
Introduction: Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disease with no known cure, limited treatment options with minimal benefits, and significant unmet need for disease modifying therapies.
Aims: This study investigated memantine's impact on ALS progression, with an additional focus on the effects of memantine on cognitive and behavioral changes associated with the disease.
Methods: A randomized, double-blind, placebo-controlled clinical trial was conducted from December 2018 to September 2020.
Amyotrophic lateral sclerosis (ALS) patients lack effective treatments to maintain motor and neuromuscular function. This study aimed to evaluate the effect of a home-based exercise program on muscle strength, ALS scores, and transcriptome in ALS patients, Clinical Trials.gov #NCT03201991 (28/06/2017).
View Article and Find Full Text PDFA protocol for scoping reviews on the role of whole-body and dedicated body-part magnetic resonance imaging for assessment of adult and juvenile idiopathic inflammatory myopathies.
Rheumatol Int
November 2024
Article Synopsis
- * The review aims to gather and assess existing MRI scoring systems to create an evidence-based foundation for a universal standardized system that can be used in both research and clinical settings.
- * A systematic search of electronic databases will be conducted to consolidate information on MRI scanning protocols for evaluating muscle involvement in IIMs, with the goal of producing guidelines for consistent clinical and research practices.
Background And Purpose: Various electrodiagnostic criteria have been developed in Guillain-Barré syndrome (GBS). Their performance in a broad representation of GBS patients has not been evaluated. Motor conduction data from the International GBS Outcome Study (IGOS) cohort were used to compare two widely used criterion sets and relate these to diagnostic amyotrophic lateral sclerosis criteria.
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