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Drug Development

A total of 291 posts are filed under Drug Development
Abeona Expands Pipeline with Addition of Cystic Fibrosis Gene Therapy
Rare Daily Staff Abeona Therapeutics said it expanded its pipeline with the addition of ABO-401, an experimental gene therapy for cystic… Continue Reading
U.S. Food and Drug Administration Orphan Drug Designations in November 2018
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FDA Grants IntraBio Pediatric Disease Designation for Experimental Niemann-Pick Disease Therapy
Rare Daily Staff The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to IntraBio’s  lead compound series… Continue Reading
FDA Approves Catalyst’s LEMS Drug
Rare Daily Staff The U.S. Food and Drug Administration today said it approval Catalyst Pharmaceuticals’ Firdapse, the first treatment for… Continue Reading
Vertex Reports Positive, Interim Late-Stage Results from Triple Combo Therapy for CF
Rare Daily Staff Vertex Pharmaceuticals said that treatment with a triple combination of its next-generation corrector VX-659, tezacaftor,… Continue Reading
Now Comes the Hard Part
Earlier this month during Novartis’ R&D Day, Dave Lennon caught the attention of the payer community when he said that the company’s… Continue Reading
FDA Approves Sobi’s Gamifant as the First and Only Treatment Primary HLH
Rare Daily Staff The U.S. Food and Drug Administration approved Gamifant, an interferon gamma blocking antibody for the treatment primary… Continue Reading

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