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Drug Development

A total of 123 posts are filed under Drug Development
FDA Grants Modus Therapeutics’ Sevuparin Rare Pediatric Disease Designation for Treatment of Sickle Cell Disease
Rare Daily Staff The U.S. Food and Drug Administration granted Modus Therapeutics’ sevuparin Rare Pediatric Disease designation for the… Continue Reading
NEJM Publishes Promising Bluebird Bio Gene Therapy Data in Beta Thalassemia
Rare Daily Staff Interim data published in the New England Journal of Medicine from two separate studies of Bluebird Bio’s LentiGlobin,… Continue Reading
FDA Clears for Commercial Use Exemplar Genetics’ Animal Model for Rare Neurodegenerative Disease
Rare Daily Staff The U.S. Food and Drug Administration cleared Intrexon subsidiary Exemplar Genetics’ ExeGen ATM MiniSwine model for… Continue Reading
Ultragenyx and Kyowa Kirin Win FDA Approval for Crysvita to Treat XLH
Rare Daily Staff The U.S. Food and Drug Administration granted approval to Crysvita, Ultragenyx and Kyowa Kirin International’s treatment… Continue Reading
FDA Approves Rigel’s Treatment for Chronic Immune Thrombocytopenia (ITP) in Adult Patients
Rare Daily Staff The U.S. Food and Drug Administration approved Rigel Pharmaceuticals’ Tavalisse for the treatment of adults with chronic… Continue Reading
EC Approves Kineret To Treat Still’s Disease
Rare Daily Staff The European Commission expanded the approved indications for Swedish Orphan Biovitrum’s Kineret to include the treatment… Continue Reading
FDA Approves Novartis’ Afinitor DISPERZ to Treat TSC-Associated Partial-Onset Seizures
Rare Daily Staff     The U.S. Food and Drug Administration approved Novartis’ Afinitor DISPERZ for the adjunctive treatment of adult… Continue Reading