October 14, 2019
The Mother of Invention Strikes Again
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October 8, 2019
FDA Awards 12 Grants to Fund Rare Disease Therapy Clinical Trials
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October 8, 2019
FDA Provides $4.1 Million for Two Natural History Studies
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September 23, 2019
FDA Places Hold on Abeona Ahead of Pivotal Trial of Cell Therapy for RDEB
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September 6, 2019
FDA Clarifies Accelerated Pathway for Medical Devices for Rare Disease Indications
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September 6, 2019
Takeda Issues Recall for Natpara
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August 29, 2019
Report of Patient Deaths Creates Questions about Vertex’s CF Drug
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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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