Seeking Answers for Undiagnosed Patients

For patients with a rare disease, the diagnostic…

Shire Rejects Takeda’s $61.9 Billion Bid as Allergan Says It May Jump into the Fray
Rare Daily Staff Rare disease drugmaker Shire said it has rejected a $61.9 billion takeover bid from from Japanese drug giant Takeda as… Continue Reading
ICER Says Roche’s Hemophilia A Therapy Improves Health While Lowering Costs
Rare Daily Staff The Institute for Clinical and Economic Review, an independent, nonprofit that analyzes the cost-effectiveness of… Continue Reading
Why Patient Groups Shouldn’t Dismiss Report on Pharma Influence
Earlier this month, Kaiser Health News launched “Pre$cription for Power,” a database that tracks Big Pharma contributions to patient… Continue Reading
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
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
Shire Announces Sale of Oncology Business to Servier for $2.4 Billion
Rare Daily Staff Shire said it had sold its oncology business to Servier for $2.4 billion. The sale comes as Takeda has acknowledged that it… Continue Reading