Ten children with an especially rare and hard-to-treat form of "bubble boy" disease are living normal lives after receiving a new gene therapy approach, researchers say. Experts said the findings are ...

UCLA researchers are restarting a study to give a gene therapy to kids with an ultra-rare disease. While the one-time therapy is safe and effective, the drug industry has been uninterested. The ...

Later this spring, a little girl in California who essentially has no immune system will receive a lifesaving treatment for “bubble boy disease” thanks to the persistence of a dogged group of parents, ...

“I remember asking the doctor if my daughter was going to die,” said Chelsea Oakley, mother of Cora Oakley, a newborn who was diagnosed with ADA-SCID when she was just seven days old, in April 2017.

(L to R) First author Koon-Kiu Yan, Ph.D., St. Jude Department of Computational Biology, (seated) co-corresponding Stephen Gottschalk, M.D., St. Jude Department of Bone Marrow Transplantation and ...

Ten young children born without functioning immune systems and lacking the ability to fight infections are on track for healthier lives thanks to a new gene therapy treatment. Ten young children born ...

UCSF Pediatrics Professor Jennifer Puck, MD, has spent decades pushing for universal newborn screening to detect severe combined immune deficiency (SCID), a rare but deadly genetic disorder. Today, ...

He ate, played and learned like any other kid. But David Vetter’s life unfolded in a series of unusual environments: plastic, bubblelike enclosures that protected him from germs. He had severe ...

Building on its contributions to newborn screening globally, PerkinElmer’s latest FDA authorized assay enables the simultaneous detection of SMA and SCID SMA is a leading genetic cause of infant death ...