Application
A class of drugs that inhibit molecules responsible for the severe nervous system complications associated with myotonic dystrophy.
Key Benefits
- Potentially the first medication for treating the severe nervous system symptoms associated with myotonic dystrophy by targeting GABRG2.
- Transdermal and oral administration.
- May drive the development of new therapies that target GABRG2.
Market Summary
Myotonic dystrophy (DM) is considered a subgroup of myopathy and the most common type of muscular dystrophy. While DM commonly presents as an adult-onset multisystem degenerative disorder, it may also affect fetal and postnatal growth in individuals with significant expansions. Clinical presentation is diverse and ranges from asymptomatic electrical myotonia to severe weakness and disability, including cardiac conduction defects, infertility, cataracts, and insulin resistance. There is no cure or specific treatment for myotonic dystrophy; however, ankle support and leg braces can help when muscle weakness worsens. In addition, some medications and procedures can lessen myotonia, heart, and cataracts.
Technical Summary
Researchers at Emory have identified competitive antagonists that inhibit gamma-aminobutyric acid type A receptor (GABRG2) and endozepine-like molecules for treating the nervous system complications associated with DM. These undesired effects include hypersomnia, anhedonia, impaired cognition, executive function, reduced alertness, motivation, and arousal. The inventors treated a patient with the antagonists, and the patient indicated they experienced reduced sleep per day, and naps were more “refreshing.” In addition, the individual displayed multiple phenotypic improvements.
Developmental Stage
Early-stage with human data.