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
ProQR, Rhythm Complete Public Offerings
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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
AI Drug Developer Healx Secures $56 million to Launch Global Rare Disease Accelerator Program
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October 16, 2019
Editas and AskBio Team Up to Develop Novel Treatments for Neurological Diseases
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October 16, 2019
Ipsen Expands Its Rare Disease Portfolio with License for Blueprint Medicines’ FOP Treatment
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October 16, 2019
Alexion Adds to Rare Disease Pipeline with Acquisition of Achillion
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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
Roche Said Study Shows Its Therapy Is Superior to Commonly Used Treatment in Rare Autoimmune Disease
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