A few drops of blood from a newborn can reveal so much about the health of a baby. The testing of those blood droplets, along with tests for hearing loss and critical congenital heart conditions, comprise a routine newborn screening. And while the hearing test and heart test are relatively simple evaluations, the blood screening is more complex
Read MoreWhile treatment for severe combined immunodeficiency (SCID) can restore a person's immune system, the procedure doesn’t signal an end to coping with this life-threatening PI. A reconstituted immune system may fail, and side effects from the treatment may present lifelong challenges. Those with SCID must monitor their immune system and general
Read MoreOrchard Therapeutics recently announced that it would stop using Strimvelis, a gammaretroviral vector-based gene therapy, to treat persons with ADA-SCID (adenosine deaminase deficiency severe combined immunodeficiency). The company made the decision because a patient treated in 2016 with Strimvelis has developed leukemia, which could be
Read MoreWhile the Primary Immune Deficiency Treatment Consortium (PIDTC) is a multi-faceted organization with many moving parts, one thing is certain – its goal is to improve the lives of those with rare, life-threatening immune system disorders. Dr. Jennifer Puck, principal investigator for the PIDTC and professor of pediatrics at University of
Read MoreSeven-year-old Ava George, who has severe combined immunodeficiency (SCID), and her parents – Erica Billy and Terrance George – are one of several families highlighted in a recently released documentary film series, “Ending Disease: The Stem Cell, Anti-Cancer T-Cell, & Antibody Revolution in Medicine.” “Ending Disease” is a four-part film
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