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Flumazenil for Treatment of CNS Symptoms in Myotonic Dystrophy
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...
Published: 4/23/2024       Contributor(s): Gary Bassell, Andrew Jenkins, David Rye, Eric Wang, Maurice Swanson, Lyndon Lien
MicroRNAs (miRNAs) for the Treatment of Neurological Disorders
Application Oligonucleotides that target Kv4.2 miRNAs for the upregulation of Kv4.2 protein in neurological disorders. Key Benefits Increases the abundance of the neuro-protective protein, Kv4.2. Offers a potential treatment for neurological diseases, including Fragile X syndrome, autism spectrum disorders, epilepsy, and Alzheimer's disease. Market...
Published: 4/23/2024       Contributor(s): Gary Bassell, Xiaodi Yao, Christina Gross