Ionis Launches Phase 1 Trial of IONIS-DMPK Rx to Treat DM1

Ionis Pharmaceuticals, Inc. (formerly Isis Pharmaceuticals, Inc.) announced today that it has launched a Phase 1 clinical trial for IONIS-DMPKRX. IONIS-DMPKRX is designed to reduce the production of toxic dystrophia myotonic-protein kinase (DMPK) RNA in cells, including muscle cells, for the treatment of Myotonic Dystrophy Type 1 (DM1).

Genetic Testing for Myotonic Dystrophy

Myotonic community members often contact the Foundation with questions about genetic testing. We spoke with Carly Siskind of Stanford's Neuromuscular Team about the pros and cons of testing.

The Mef2 Transcription Network is Disrupted in DM Heart Tissue

Researchers at important academic labs around the US have recently published exciting new information about advances in DM research.

Diagnostic Odyssey of Patients with Myotonic Dystrophy, Journal of Neurology, 2013

Researchers from the University of Rochester recently summarized the “diagnostic odyssey” experienced by a group of 814 individuals with myotonic dystrophy.

Impact of Childhood and Congenital DM on Quality of Life

Nicholas Johnson, MD, and researchers at the University of Rochester recently published an article in The Journal of Child Neurology that describes the impact of childhood and congenital myotonic dystrophy on quality of life.

A New Study Provides Hope for DM Treatments

Maurice Swanson, Ph.D., Professor of Molecular Genetics and Microbiology at University of Florida, Gainesville, and a team of researchers have found that the muscleblind-like 2 (MBNL2) protein in the central nervous system (CNS) may be responsible for the neurological impacts of myotonic dystrophy

Dysregulation of Circular RNAs in DM1

Circular RNAs that play a role in myogenesis are dysregulated in DM1 skeletal muscle.

What is the Actual Progenitor Allele Length in Any Given DM1 Patient?

A new study examines approaches to determine DM1 study subject progenitor allele length.

Targeting DM1 with a Multivalent Ligand

Multivalent ligands show bioavailability and efficacy in targeting splicing defects in DM1.

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