Last Updated: 09/26/2026
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Found 11 publications
Analysis of human urinary extracellular vesicles reveals disordered renal metabolism in myotonic dystrophy type 1.
Journal: Nature communications
Published: December 19, 2023
Correction of Clcn1 alternative splicing reverses muscle fiber type transition in mice with myotonic dystrophy.
Journal: Nature communications
Published: September 04, 2022
Antisense oligonucleotide and adjuvant exercise therapy reverse fatigue in old mice with myotonic dystrophy.
Journal: Molecular therapy. Nucleic acids
Published: July 24, 2020
Urine mRNA to identify a novel pseudoexon causing dystrophinopathy.
Journal: Annals of clinical and translational neurology
Published: March 08, 2019
Analysis of extracellular mRNA in human urine reveals splice variant biomarkers of muscular dystrophies.
Journal: Nature communications
Published: December 06, 2017
Targeting DMPK with Antisense Oligonucleotide Improves Muscle Strength in Myotonic Dystrophy Type 1 Mice.
Journal: Molecular therapy. Nucleic acids
Published: October 27, 2016
Case Records of the Massachusetts General Hospital. Case 30-2015: A 50-Year-Old Man with Cardiogenic Shock.
Journal: The New England journal of medicine
Published: September 24, 2015
Identification and characterization of modified antisense oligonucleotides targeting DMPK in mice and nonhuman primates for the treatment of myotonic dystrophy type 1.
Journal: The Journal of pharmacology and experimental therapeutics
Published: June 18, 2015
Splicing biomarkers of disease severity in myotonic dystrophy.
Journal: Annals of neurology
Published: June 04, 2013
RNA interference targeting CUG repeats in a mouse model of myotonic dystrophy.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: November 28, 2012
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Last Updated: 09/26/2026