| 1 |
2014 FDA drug approvals. Nat Rev Drug Discov. 2015 Feb;14(2):77-81.
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| 2 |
Clinical pipeline report, company report or official report of Biogen idec.
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| 3 |
ClinicalTrials.gov (NCT03569891) HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients. U.S. National Institutes of Health.
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| 4 |
ClinicalTrials.gov (NCT05135559) Open-label Study Investigating Efficacy, Safety and Pharmacokinetics of Concizumab Prophylaxis in Children Below 12 Years With Haemophilia A or B With or Without Inhibitors. U.S.National Institutes of Health.
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| 5 |
ClinicalTrials.gov (NCT03861273) A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B (BENEGENE-2). U.S. National Institutes of Health.
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| 6 |
Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
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| 7 |
Clinical pipeline report, company report or official report of OPKO Health
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| 8 |
ClinicalTrials.gov (NCT01687608) Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B. U.S. National Institutes of Health.
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| 9 |
ClinicalTrials.gov (NCT02695160) Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B. U.S. National Institutes of Health.
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