August 19, 2019
FDA Approves Celgene’s Treatment for Rare Bone Cancer
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August 19, 2019
FDA Approves Genentech’s Rozlytrek to Treat Two Rare Cancers
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August 16, 2019
Tackling Kidney Diseases with Orally-Delivered Enzymes
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August 15, 2019
Ultragenyx Enters Partnership with GeneTx to Develop Angelman Syndrome ASO
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August 13, 2019
Pharming Acquires Late-Stage Novartis Drug to Treat Ultra-Rare Immune Disorder
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August 12, 2019
ProQR Gets FDA Clearance to Start Clinical Trial to Treat Rare Eye Disease
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August 12, 2019
Immunic Says First Patient Enrolled in Proof-of-Concept Trial for Rare Liver Disease Therapy
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August 9, 2019
Giving Voice to Patients through Data
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August 9, 2019
Sarepta Says DMD Gene Therapy Adverse Event Report Submitted Erroneously
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August 7, 2019
Akcea’ and Ionis’ Waylivra Meets Endpoints in Study in Patients with FPL
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August 7, 2019
Rhythm Reports Positive Pivotal Results from Rare Obesity Trials
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August 7, 2019
Critical Path Institute and NORD Launch Rare Disease Data Platform
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