Isis Pharmaceuticals has announced the opening of a U.S. trial to evaluate the safety and tolerability of ISIS-DMPKRx in adults with type 1 myotonic dystrophy
A phase 1-2 trial of ISIS-DMPKRx, an experimental compound for the treatment of type 1 myotonic muscular dystrophy, is now underway in the U.S. in adults with this disorder who are 20 to 55 years old and meet other study criteria.
Isis Pharmaceuticals is launching a phase 1 trial of ISIS-DMPKRx, an experimental drug for type 1 myotonic muscular dystrophy
Update (June 11, 2014): This story has been updated with additional information about the phase 1 trial of ISIS-DMPKRx in healthy volunteers and the planned trial in people with type 1 MMD, as well as the availability of a fact sheet on this drug provided by Isis Pharmaceuticals...
Abnormalities in the muscle enzyme pyruvate kinase contribute to type 1 myotonic dystrophy, an MDA-funded study has found
An abnormality in a muscle enzyme known as pyruvate kinase (PKM) has been added to the list of things that go wrong in type 1 myotonic muscular dystrophy (MMD1, or DM1), a new study has found.
MDA supported Thomas Cooper at Baylor College of Medicine in Houston on this study, which was published online in Proceedings of the National Academy of Sciences July 30, 2013.
Small molecules that induce or reverse effects of the type 1 myotonic dystrophy mutation in cells in the lab are likely to speed research
Matthew Disney is a current and former MDA grantee at the Scripps Research Institute in Jupiter, Fla. Disney's current MDA grant is focused on targeting toxic RNA in type 2 myotonic dystrophy (MMD2, or DM2).
Adding a cell-penetrating peptide to a compound that reduces clump formation in cell nuclei increased its effectiveness in type 1 myotonic dystrophy research mice
Scientists at the biopharmaceutical company Genzyme, working with mice, say they have modified and improved an existing experimental strategy to treat type 1 myotonic muscular dystrophy (MMD1, also known as DM1).
Located around the United States, MDA’s new network provides a foundation for the conduct of clinical trials in MMD1 and MMD2
MDA has launched a new, five-center clinical research network focused on type 1 and type 2 myotonic muscular dystrophy (MMD1 and MMD2, also known as DM1 and DM2), with the principal goal of preparing for testing of new MMD treatments as they become available.
Treatment with IGF1 improved motor function, slowed weight loss, improved muscle health and increased survival time in mice with a disease resembling spinal-bulbar muscular atrophy
Mice with a disease resembling spinal-bulbar muscular atrophy (SBMA, or Kennedy disease) that were treated with a compound based on insulin-like growth factor 1 (IGF1) had better motor function, slower weight loss, healthier muscles and longer survival time than mice that received an inactive substance, an MDA-supported research team has reported.