Muscular dystrophy - Open Access .click

Results 101 to 110 of about 78,813 (265)

Assessing the socio-economic burden of inherited and inflammatory neuromuscular diseases (BIND study): a study protocol

Orphanet Journal of Rare Diseases
Introduction Neuromuscular diseases (NMDs) are rare multisystem, genetic or acquired disorders causing weakness and/or sensory loss. It is essential for governments, insurance providers, and broader society to have a better understanding of the burden of
Ian C. Smith +14 more
doaj +1 more source

Reachable Workspace as a Clinical Outcome for Upper Extremity Function: A Narrative Review

Muscle &Nerve, EarlyView.
ABSTRACT Motion sensing technology can be utilized to capture detailed upper extremity (UE) motion to reconstruct an individual's three‐dimensional (3D) reachable workspace (RWS). The RWS can be quantified as relative surface area (RSA), providing an innovative surrogate measure to assess UE mobility and function.
Jay J. Han +3 more
wiley +1 more source

Gait analysis reveals new outcome measures for monitoring disease progression in individuals with late-onset Pompe disease

Journal of NeuroEngineering and Rehabilitation
Background Late-onset Pompe disease (LOPD) presents with progressive muscle weakness, often leading to functional impairment that is challenging to monitor with conventional assessments.
Mireia Claramunt-Molet +12 more
doaj +1 more source

No Evidence for an Association Between DIP2B Repeat Expansion and Neurological Disease

Movement Disorders, EarlyView.
Chia‐Ying Ko +9 more
wiley +1 more source

Treatment Preferences of Patients With Myasthenia Gravis: A Qualitative Study

Muscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims The burden of myasthenia gravis (MG) is often underestimated, and studies usually focus on the symptom burden. However, treatment‐related adverse events also contribute to patients' burdens and affect their treatment decisions.
Meg Mendoza +6 more
wiley +1 more source

Exploring the Content Validity of Patient‐Reported Outcome Measures to Capture the Patient Experience of Becker Muscular Dystrophy

Muscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims The patient experience of Becker muscular dystrophy (BMD) is not well understood, making it difficult to evaluate the conceptual relevance of proposed patient‐reported outcome (PRO) measures. This study aimed to conceptualize the patient experience of BMD and evaluate content validity and perceptions of meaningful changes of ...
Abby Bronson +6 more
wiley +1 more source

Optimizing Research Operations and Resource Utilization in ALS Care: Insights From the Tofersen Antisense Oligonucleotide Expanded Access Protocol

Muscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims Tofersen is a gene‐targeted therapy for individuals with superoxide dismutase 1 (SOD1) (+) amyotrophic lateral sclerosis (ALS). Prior to U.S. Food and Drug Administration (FDA) approval, tofersen was made available through expanded access protocol.
Alison Wheeler +30 more
wiley +1 more source

At‐Home Versus in‐Clinic Vital Capacity Measurement: Insights From the HEALEY ALS Platform Trial

Muscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims Respiratory weakness, typically monitored as vital capacity (VC), is a central feature of amyotrophic lateral sclerosis (ALS). VC is increasingly measured remotely in participants' homes, although in‐clinic assessment remains the standard.
Eric A. Macklin +269 more
wiley +1 more source

The Use of the Four Square Step Test and the 10‐m Walk/Run Test to Determine Fall Risk in Children With Duchenne Muscular Dystrophy

Muscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims The identification of the risk of falling in Duchenne muscular dystrophy (DMD) is essential for the implementation of timely preventive approaches. This study aimed to examine the ability of the four square step test (FSST) and the 10‐m walk/run test (10MWRT) to discriminate between fallers and non‐fallers in children with ...
Numan Bulut +3 more
wiley +1 more source

Muscle Strength, Balance, and Indoor Mobility in Oculopharyngeal Muscular Dystrophy: An Exploratory Canadian Multicenter Study

Muscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims Oculopharyngeal muscular dystrophy (OPMD) is a neuromuscular disease presenting with dysphagia, ptosis, and proximal weakness. Muscle strength and indoor mobility capacity have rarely been studied using standardized clinical outcome assessments (COAs). The objectives of this study were to (1) document muscle strength, balance,
Nicolas Bélair +11 more
wiley +1 more source

humans
muscular dystrophies
3. good health

child
male
animals

dystrophin
muscular dystrophy, facioscapulohumeral
duchenne muscular dystrophy