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).
A recently published study from Sweden reported impaired facial recognition in people with DM1 and indicated that there are brain differences that affect how faces are perceived and stored by people with DM1.
MDF 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.
Researchers at important academic labs around the US have recently published exciting new information about advances in DM research.
Researchers have published several recent studies on advances in DM research.