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| Praxis Precision Medicines, Inc.: Praxis Precision Medicines Announces Extension Period for Relutrigine for Treatment of SCN2A and SCN8A Developmental and Epileptic Encephalopathies | FDA sets updated PDUFA date of December 27, 2026 BOSTON, June 29, 2026 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a fully integrated, leading central nervous system (CNS)... ► Artikel lesen |
| Praxis Precision Medicines, Inc.: Praxis Precision Medicines Provides Vormatrigine Program Update | POWER1 Study in highly refractory patients with focal onset seizures did not meet its primary success measure Secondary measure, the 50% response rate, was met and seizure reduction during the second... ► Artikel lesen |
| Praxis Precision Medicines, Inc.: Praxis Precision Medicines Provides Corporate Update and Reports First Quarter 2026 Financial Results | FDA accepted the new drug application (NDA) for ulixacaltamide in Essential Tremor with a PDUFA target action date of January 29, 2027, and the NDA for relutrigine, with priority review, in SCN2A... ► Artikel lesen |
| Disc Medicine Inc: Disc Medicine Reports Second Quarter 2026 Financial Results and Provides Business Update | Completed Type A meeting with the FDA on bitopertin and aligned that the Phase 3 APOLLO study, if successful, can serve as the basis for CRL response; topline data expected in Q4 2026Launched Expanded... ► Artikel lesen |
| Disc Medicine Inc: Disc Medicine Provides Update from FDA Type A Meeting for Bitopertin in Erythropoietic Protoporphyria | Aligned with the FDA that the Phase 3 APOLLO study, if successful, can serve as the basis for CRL response and could potentially support a traditional approvalOn track for expected CRL response submission... ► Artikel lesen |
| Beam Therapeutics Presents Updated Clinical Data from the Phase 1/2 Trial of BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) at the European Respiratory Society (ERS) Congress 2026 | BEAM-302 Continues to Demonstrate Potential as One-time Treatment for Both Lung and Liver Manifestations of AATD, with AAT Protein Production Under Normal Physiological Control With Up to 18 Months... ► Artikel lesen |
| Beam Therapeutics Reports Second Quarter 2026 Financial Results and Announces First Patient Dosed in Global Pivotal Cohort of BEAM-302 Trial in Alpha-1 Antitrypsin Deficiency (AATD) | Updated BEAM-302 Phase 1/2 Clinical Data Selected for Late-Breaking Oral Presentation at the European Respiratory Society (ERS) Congress 2026 Dosing Complete for All Adult and Adolescent Patients... ► Artikel lesen |
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