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SKYSONA
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Skysona
(ELIVALDOGENE AUTOTEMCEL)Orphan drugacceleratedGenetix Biotherapeutics Inc.
12.1 Mechanism of Action SKYSONA adds functional copies of the ABCD1 cDNA into patients' hematopoietic stem cells (HSCs) through transduction of autol...
Paul Orchard, MD, MD
University of Minnesota Medical Center
📍 MINNEAPOLIS, MN
Florian Eichler, MD, MD
Massachusetts General Hospital
📍 BOSTON, MA
Wolfgang Köhler, MD
Leipzig University Medical Center, Leukodystrophy Outpatient Clinic, Department of Neurology, Leipzig, Germany
Florian S Eichler, MD
Massachusetts General Hospital
📍 Boston, Massachusetts
Adeline Vanderver, MD
Children's Hospital of Philadelphia
📍 Los Angeles, California
Mitchell S Cairo, MD, MD
New York Medical College
📍 HAWTHORNE, NY
View all X-linked cerebral adrenoleukodystrophy specialists →