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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October 31, 2019
Catalyst Firdapse Fails in Congenital Myasthenic Syndromes Trial
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October 25, 2019
Cabaletta Bio Raises $75 Million in IPO for T-Cell Therapies for Autoimmune Diseases
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October 23, 2019
Moderna Receives FDA Fast Track Designation for Propionic Acidemia Program
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October 10, 2019
Alexion Pays Stealth $30 Million for Option to Co-Develop Late-Stage Program in Mitochondrial Diseases
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October 8, 2019
FDA Approves First Treatment to Increase Pain-Free Light Exposure in Rare Disorder
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October 8, 2019
Avrobio Doses First Patient in Gene Therapy Trial for Cystinosis
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October 4, 2019
Sarepta Therapeutics Reports Positive Results for Limb-Girdle Gene Therapy
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October 4, 2019
Nippon Shinyaku Files for Approval of DMD Exon-Skipping Drug
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