January 20, 2020
FDA to Move to Online Submissions for Orphan Drug Designation Requests
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January 3, 2020
NICE Rejects Akcea’s FCS Drug Waylivra as Too Costly
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December 31, 2019
A Down Year for Drug Approvals, But Notable for Its Standouts
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December 31, 2019
Complete FDA Orphan Drug Approvals in 2019
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November 22, 2019
Addressing the Barriers to Accessing Experimental Therapies
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November 18, 2019
FDA Grants Anavex Rare Pediatric Disease Designation
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November 18, 2019
EMA Grants Orchard Accelerated Assessment of Therapy for Patients with MLD
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October 31, 2019
FDA Places Partial Hold on Trials of Zolgensma Injected into Spinal Fluid
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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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