June 29, 2017
CheckRare: FDA Approves Haegarda to Treat HAE
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June 23, 2017
Rare Disease Report: RASRx is the Latest Duchenne Drug To Get Orphan Status From the FDA
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June 22, 2017
Gottlieb’s Aggressive Plans to Streamline the Orphan Drug Review Process
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June 21, 2017
Rare Disease Patient and Lawyer: “I Want to Walk Again”
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June 5, 2017
ProQR Receives Fast Track Designation from the FDA for QR-110 for Leber’s Congenital Amaurosis Type 10
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May 24, 2017
FDA Grants Priority Review Status to Ultragenyx
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May 16, 2017
NYT: Senate Confirms Scott Gottlieb to Head F.D.A.
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May 1, 2017
Horizon Pharma plc Announces FDA Approval to Expand the Age Range for RAVICTI® to People with Urea Cycle Disorders Ages Two Months +
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April 29, 2017
Scott Gottlieb Nomination Approved
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April 24, 2017
BioPharma Dive: 5 Trends Shaping Rare Disease Drug Development
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April 23, 2017
Designing Effective Rare Disease Registries
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April 9, 2017
Teva Announces FDA Approval of AUSTEDO™ (deutetrabenazine) Tablets for the Treatment of Chorea Associated with Huntington’s Disease
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