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Efficacy of Muscle Exercise in Patients with Muscular Dystrophy
Myotonic dystrophy type 1 is a neuromuscular disorder affecting multiple organ systems and is characterized by a variety of clinical ...
Dosage effect of multiple genes accounts for multisystem ... - bioRxiv
DM1 is an uncommon cause of hypoxia due to weakness of the respiratory muscles leading to alveolar hypoventilation. Key physical examination ...
Gastrointestinal manifestations in myotonic muscular dystrophy - ARPI
Cell and animal models of DM1 have been crucial to providing insight into disease mechanisms and to revealing new therapeutic targets.
Protocole National de Diagnostic et de Soins (PNDS) Dystrophie ...
Recent achievements carried out on lipid nanoparticles enabling gene therapy with the approval of treatment for rare disease as Onpattro® and ...
Molecular Therapies for Myotonic Dystrophy Type 1 - CNR Iris
Since a curative treatment for DM1 is not yet available, patient management also requires frequent screening for possible disease complications.
Drug delivery nanosystems for skeletal muscle treatment in myotonic ...
Patients reported limited benefits from the symptomatic therapies available. They hope for a treatment that will halt or slow disease ...
Myotonic dystrophy type 1 | Maastricht University
Increased muscleblind levels by chloroquine treatment improve myotonic dystrophy ... Leptin and the metabolic syndrome in patients with myotonic dystrophy type 1.
Voice of the Patient report - Myotonic Dystrophy Foundation
New nomenclature and DNA testing guidelines for myotonic dystrophy ... repeat in the blood DNA of myotonic dystrophy type 1 patients. DNA.
Therapeutic potential of oleic acid supplementation in myotonic ...
DM1 is an autosomal dominant inherited disease that represents the most common form of muscular dystrophy in adults with a prevalence of 1 in 8,000 individuals ...
Myotonic Dystrophy Type 1 (DM1): From the Genetics to Molecular ...
ABSTRACT: Introduction: Myotonic dystrophy (DM) is a chronic, multisystemic, neurological condition. Patients and caregivers are.