January 24, 2020
FDA Approves First Treatment for Rare Soft Tissue Cancer
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January 22, 2020
Making the Value Proposition for Rare Disease Therapies
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January 13, 2020
Ultragenyx Reports Positive Topline Results from OTC Deficiency Gene Therapy Study
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January 7, 2020
Deep Genomics Raises $40 Million in Series B Financing
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January 7, 2020
Rhythm CEO to Step Down After FDA Filing
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January 6, 2020
FDA Grants QED Fast Track and Orphan Drug Designations for Rare Cancer Therapy
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December 17, 2019
Triplet Therapeutics Launches with $59 Million to Develop Treatments for Repeat Expansion Disorders
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December 10, 2019
Sanofi Presents Positive Results from Pivotal Trial of Sutimlimab in people with CAD
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December 2, 2019
Neurocrine and Xenon Partner to Develop Treatment for Rare Epilepsy
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November 27, 2019
La Jolla Pharmaceuticals Halts Development of Beta Thalassemia Drug as CEO Departs
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November 21, 2019
FDA Approves Alnylam’s RNAi Drug Givlaari for AHP Attacks
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November 19, 2019
Gene Editing Treatment Shows Positive Results in First Two Treated Patients with Blood Disorders
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