Amsterdam, Netherlands – Findings from a new study in the Journal of Neuromuscular Diseases, published by IOS Press, demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy (SMA). The study’s results add further support for gene therapy as a treatment modality that can deliver durable transformative...
Latest News
OSAKA, Japan & CAMBRIDGE, Mass. – VPRIV (velaglucerase alfa), an approved enzyme replacement therapy (ERT) for Gaucher disease, appears to be safe and effective in children under 4—a population not included in the clinical trials that supported its approval. A recent U.S. real-world study evaluated VPRIV in a small group...
NORFOLK, Va. — ReAlta Life Sciences, Inc., a mid-stage clinical biotech company dedicated to saving lives by rebalancing the inflammatory response to address life-threatening diseases, today announced enrollment of the first patient in a Phase 2 clinical trial evaluating the safety and efficacy of RLS-0071, an investigational new drug based...
NORFOLK, Va. — ReAlta Life Sciences, Inc. (“ReAlta” or the “Company”), a clinical-stage biopharmaceutical company dedicated to saving lives by rebalancing the inflammatory response to address rare and acute inflammatory diseases, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to RLS-0071 (pegtarazimod) for the treatment of...
PLANO, Texas – Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” or “we”), a clinical-stage biopharmaceutical company, today announced that it received a communication from the Division of Neurology Products 1 (“Division”) of the U.S. Food and Drug Administration (“FDA”) stating that, after a preliminary review of briefing materials for...
PLANO, Texas – Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” or “we”), a clinical-stage biopharmaceutical company, today announced that it has submitted a New Drug Application (“NDA”) for bardoxolone methyl (“bardoxolone”) for the treatment of chronic kidney disease (“CKD”) caused by Alport syndrome to the U.S. Food and Drug...
Shares of Reata Pharmaceuticals Inc (RETA.O) tripled in value on Wednesday after the U.S. Food and Drug Administration (FDA) approved its rare disease drug, ending years of uncertainty over its future.
MENLO PARK, Calif. — ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted clearance for the initiation of Part 3 of its ongoing Phase 2 clinical...
BRIDGEWATER, N.J. — Recordati Rare Diseases Inc. today announced the presentation of new data detailing advances in the understanding of Castleman disease (CD), a group of rare immune system disorders characterized by abnormal lymph node enlargement and systemic inflammatory symptoms that can be potentially life-threatening. In a series of poster presentations...
MILAN – Italian pharmaceutical group Recordati (RECI.MI) has signed an agreement to buy UK-headquartered EUSA Pharma for an enterprise value of 750 million euros ($847 million) to boost its rare disease products portfolio, it said in a statement on Friday. EUSA Pharma, founded in 2015 and controlled by funds managed by...