Rett Syndrome
Oct 07, 2025
Aminex Therapeutics Secures FDA Orphan Drug Designation for AMXT 1501 + DFMO in Neuroblastoma; Eccogene Gets FDA Nod to Begin Phase IIa MOSAIC Trial of ECC4703 + ECC0509 in MASH; Orca Bio’s Orca-T Earns FDA Priority Review for Hematologic Malignancies; Alto Neuroscience Gains FDA Fast Track for ALTO-101 in Cognitive Impairment Linked to Schizophrenia; Taysha Gene Therapies Wins FDA Breakthrough Therapy Status for TSHA-102 in Rett Syndrome
Aminex Therapeutics Secures FDA Orphan Drug Designation for AMXT 1501 + DFMO in Neuroblastoma Aminex Therapeutics, Inc., a clinical-stage biotechnology company developing novel cancer therapies, announced that the FDA has granted Orphan Drug Designation (ODD) to AMXT 1501 in combination with difluoromethylornith...
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Mar 14, 2023
Sanofi to Acquire Provention Bio; USFDA Committee Votes in Favor Roche’s Polivy; FDA to Review Mesoblast’s GvHD Drug; FDA Approves Acadia’s Daybue ; FDA Approves Nelarabine Injection; Calliditas’s Nefecon Trial Reuslt in IgA Nephropathy
Sanofi to Acquire Provention Bio for USD 2.9 Billion Sanofi and Provention Bio, Inc., a publicly traded biopharmaceutical company based in the United States focused on intercepting and preventing immune-mediated diseases such as type 1 diabetes (T1D), have agreed to acquire Provention Bio, Inc. for $25.00 per sh...
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Apr 30, 2020
Remdesivir accelerates for COVID-19 recovery; Merck pact with Almac; Pfizer, BioNTech finish dosing in; Taysha Gene Therapies for trials
Remdesivir of Gilead accelerates COVID-19 recovery Gilead Sciences' Remdesivir shows the first data from a placebo-controlled study. Remdesivir decreases recovery time for hospitalized COVID-19 patients by four days or 31% as published in a National Institutes of Health-sponsored study setting the drug against p...
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