October 22, 2019
FDA Approves Vertex’s Triple Combo Cystic Fibrosis Treatment
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October 22, 2019
Protalix Reports Positive Interim Data in Late-Stage Fabry Study
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October 21, 2019
Alexion Receives FDA Approval for Ultomiris for aHUS
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October 17, 2019
Stealth Says Experimental Drug Improves Cardiac Function in Barth Syndrome Patients
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October 16, 2019
FDA Grants ProQR Rare Pediatric Disease Designation for Rare Eye Disease Therapy
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October 15, 2019
With Rights Back in Hand, Reata Reports Positive Results for FA Therapy
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October 14, 2019
The Mother of Invention Strikes Again
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October 14, 2019
Roche Said Study Shows Its Therapy Is Superior to Commonly Used Treatment in Rare Autoimmune Disease
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October 11, 2019
Deep Genomics Boasts an AI First in Drug Discovery
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October 8, 2019
FDA Approves First Treatment to Increase Pain-Free Light Exposure in Rare Disorder
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October 8, 2019
Avrobio Doses First Patient in Gene Therapy Trial for Cystinosis
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October 7, 2019
Zogenix Presents Positive Results for Experimental Treatment for TK2 Deficiency
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