Rare Disease Report

Gene Therapy Very Effective in Hemophilia B Patients

DECEMBER 05, 2016
Frank Leebeek, MD, PhD

Frank Leebeek, MD, PhD of Erasmus MC talked to us at the 58th Annual ASH Meeting & Exposition about the data he presented showing the significant clinical breakthrough they are making with gene therapy to treat hemophilia B.


Leebeek FWG, Tangelder M, Meijer K, et al. 2314 Interim Results from a Dose Escalating Study of AMT-060 (AAV5-hFIX) Gene Transfer in Adult Patients with Severe Hemophilia B. Presented at 58th Annual ASH Meeting & Exposition; San Diego, CA; December 3-6, 2016. Abstract 2314.

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