August 13, 2019
Senate Pressure Grows on Novartis over Gene Therapy Data Manipulation
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August 9, 2019
Sarepta Says DMD Gene Therapy Adverse Event Report Submitted Erroneously
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August 7, 2019
Novartis CEO Says He’s Committed to Rebuilding Trust After Manipulation of SMA Gene Therapy Data
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July 30, 2019
FDA Updates Guidance on Rare Pediatric Disease Priority Review Vouchers
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July 5, 2019
Finding Appropriate Endpoints and Measures for Rare Eye Diseases
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July 1, 2019
FDA Orphan Drug Approvals First Half 2019
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July 1, 2019
FDA Orphan Drug Designations in Q2 2019
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June 25, 2019
FDA Tells Acer It Won’t Approve vEDS Therapy without Additional Trial
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June 12, 2019
Catalyst Sues FDA Over Approval of Competing LEMS Drug
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April 9, 2019
FDA Notifies Zogenix of Inadequacies in Dravet Syndrome Drug Application
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April 4, 2019
FDA Grants Abeona Fast Track Designation for Sanfilippo Type B Gene Therapy
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April 1, 2019
EMA Grants Krystal Biotech PRIME Designation for DEB Gene Therapy
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