Results 81 to 90 of about 253,218 (253)
Molecular Therapy: Nucleic Acids, 2019 Systemic delivery of antisense oligonucleotides (AO) for DMD exon skipping has proven effective for reframing DMD mRNA, rescuing dystrophin expression, and slowing disease progression in animal models.
Florian Barthélémy +6 more
doaj +1 more source
Magnetic Resonance in Medicine, EarlyView.ABSTRACT Purpose Spin density‐weighted (SDW) and inversion recovery (IR) 23Na MRI provide different sodium contrasts with complementary information. Therefore, the aim was to develop a time‐efficient sequence scheme capable of providing both contrasts by acquiring SDW and IR 23Na MRI data within a single sequence without additional measurement time ...
Tobias Wilferth +4 more
wiley +1 more source
, 2015 Duchenne muscular dystrophy (DMD) is the most devastating form of inherited muscular dystrophy in men. The loss of dystrophin causes progressive weakness and degeneration of skeletal and cardiac muscle leading to the replacement of muscle by connective ...
Malvestio, Lygia M. [UNESP] +3 more
core +1 more source
Outcome of Long-Term Corticosteroid Treatment in Duchenne Muscular Dystrophy
Pediatric Neurology Briefs, 2007 The clinical orthopedic effects of chronic daily corticosteroid treatment were evaluated by chart review in boys with genetically confirmed Duchenne muscular dystrophy (DMD) followed at the Ohio State University Muscular Dystrophy Clinic between 2000 and
J Gordon Millichap
doaj +1 more source
Muscle‐Specific Kinase Signaling and Its Therapeutic Potential
Muscle &Nerve, EarlyView.ABSTRACT The function of the neuromuscular junction (NMJ) is compromised in many neuromuscular diseases (NMDs) such as autoimmune or congenital myasthenia gravis (MG), amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and muscular dystrophies.
Stine Marie Jensen +2 more
wiley +1 more source
Studies on muscular dystrophy associated genes [PDF]
, 2007 Muscular dystrophy is a collective group of genetic disorder that results in progressive wasting of skeletal muscle. Dysferlin, the gene responsible for Limb Girdle Muscular Dystrophy type 2B (LGMD2B) and Miyoshi Myopathy (MM) was found to be a member of
Bakir, Hadil
core
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
PLoS ONE, 2016 Duchenne muscular dystrophy is a severe and currently incurable progressive neuromuscular condition, caused by mutations in the DMD gene that result in the inability to produce dystrophin. Lack of dystrophin leads to loss of muscle fibres and a reduction
Narinder Janghra +6 more
doaj +1 more source
A Roadmap to Newborn Screening for Duchenne Muscular Dystrophy
International Journal of Neonatal Screening, 2017 Duchenne muscular dystrophy (DMD) is the most common childhood form of muscular dystrophy, with an estimated frequency of 1:5000 live births. The impact of the disease presents as early as infancy with significant developmental delays, and ultimately ...
Samiah A. Al-Zaidy +4 more
doaj +1 more source
Therapeutics in Duchenne muscular dystrophy [PDF]
NeuroRX, 2006 Duchenne muscular dystrophy (DMD) is a fatal disorder affecting approximately 1 in 3,500 live born males, characterized by progressive muscle weakness. Several different strategies are being investigated in developing a cure for this disorder. Until a cure is found, therapeutic and supportive care is essential in preventing complications and improving ...
openaire +2 more sources