Rare Disease SCID: Boston Children’s Hospital Stem Cell Transplant Gets FDA Orphan Drug Designation

Orphan Druganaut Blog

Boston Children’s Hospital’s Division of Hematology/Oncology receives a FDA Orphan Drug Designation (ODD) January 15th, for its Stem Cell Transplant for children with the rare disease, Severe Combined Immune Deficiency X-1 (SCID-X1).

SCID prevents the immune system from fighting off diseases. Children born with SCID are usually isolated in a germ-free environment. Children with SCID must go out in the environment in “space suits” that purify their air – thus the reference to “Bubble Boy” disease.

A July 2014 journal article is published in the New England Journal of Medicine (NEJM):

Pai, S.Y., Logan, B.R., Griffith, L.M., Buckley, R.H., Parrott, R.E., Dvorak, C.C., … O’Reilly.R.J. (2014). Transplantation outcomes for severe combined immunodeficiency, 2000-2009. New England Journal of Medicine, 371(5), 434-446.

The journal article, from a research group at the Dana Farber/Boston Children’s Cancer and Blood Disorders Center, examines the implications of stem cell transplantation in children with…

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