Muscular Dystrophy Research and Tracking - CDC
WEBSep 10, 2024 · The Centers for Disease Control and Prevention (CDC) funds the Muscular Dystrophy Surveillance, Tracking, and Research Network, known as MD STARnet. MD STAR net collects critical information about muscular dystrophy that aims to improve care for those living with the disease.
Muscular Dystrophy | National Institute of Neurological …
WEBJul 19, 2024 · What is muscular dystrophy? Muscular dystrophy (MD) refers to a group of genetic diseases that cause progressive weakness and degeneration of skeletal muscles. These disorders (of which there are more than 30) vary in age of onset, severity, and the pattern of the affected muscles.
Muscular dystrophies - The Lancet
WEBMuscular dystrophies are a group of genetically inherited degenerative disorders of muscle, sharing clinical features of progressive muscle weakness and dystrophic pathological appearance on muscle biopsy. 1 In a Seminar 2 published in 2013, we reported how a better understanding of the genetic basis and mechanisms underlying these disorders has...
The Muscular Dystrophies: From Genes to Therapies
WEBThe muscular dystrophies (MDs) are a heterogeneous group of inherited disorders characterized by progressive weakness and degeneration of skeletal muscles (Table). They have traditionally been classified by clinical presentation, mode of inheritance, age of onset, and overall progression.
Muscular dystrophy: new treatments, new hopes - The Lancet
WEBNov 30, 2019 · Gene therapy for Duchenne muscular dystrophy aims to restore dystrophin concentrations by use of an adeno-associated virus (AAV) to deliver a shortened form of the dystrophin gene to muscle cells.
Duchenne muscular dystrophy - Nature Reviews Disease Primers
WEBFeb 18, 2021 · Duchenne muscular dystrophy is a severe, progressive, muscle-wasting disease that leads to difficulties with movement and, eventually, to the need for assisted ventilation and premature death.
Muscular dystrophies: causes and treatment - Nature
WEBSep 8, 2023 · This Collection brings together original research that assesses the underlying causes of muscular dystrophies, presents diagnosis and monitoring of disease progression, focuses on the...
Duchenne muscular dystrophy: disease mechanism and …
WEBJun 26, 2023 · Duchenne muscular dystrophy (DMD) is a severe, progressive, and ultimately fatal disease of skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy.
Muscular Dystrophy | Muscular Dystrophy | CDC - Centers for …
WEBMuscular dystrophies are a group of genetic disorders that result in muscle weakness over time. Each type of muscular dystrophy is different from the others.
Therapeutic approaches for Duchenne muscular dystrophy
WEBAug 31, 2023 · Abstract. Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a priority candidate for molecular and cellular therapeutics. Although rare, it is the most common...