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Praxis Precision Medicines
PRAX.US
Praxis Precision Medicines, Inc., a clinical-stage biopharmaceutical company, engages in the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance. The company’s platforms include cerebrum, a small molecule platform for CNS small molecule therapies to develop orally available precision therapies; and solidus to discover and develop antisense oligonucleotide. It also develops Ulixacaltamide, a small molecule inhibitor of T-type calcium channels that is in Phase 3 clinical trial for the treatment of essential tremor; Vormatrigine, a small molecule targeting the hyperexcitable state of sodium-channels in the brain to treat focal onset epilepsy; Relutrigine for the treatment of developmental and epileptic encephalopathies; PRAX-020 to treat KCNT1; and PRAX-050. In addition, the company develops Elsunersen, a clinical-stage ASO designed to regulate NaV1.2 expression for the treatment of SCN2A-DEE; PRAX-080 that targets PCDH19-related developmental epilepsy; PRAX-090 for targeting SYNGAP1 loss-of-function; and PRAX-100 that targets SCN2A LoF mutations.
1.945 T
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