Mutations in a handful of genes can cause severe combined immunodeficiency (SCID), which severely disrupts the body's ability to fight infection. The condition is typically fatal in the first year of ...

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, ...

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 pioneered at UC San ...

He came to UAB from Baylor University, where in 1971 he assisted in the implementation of the first germ-free environmental bubble in the United States for a patient born with severe combined ...

Researchers headed by teams at Newcastle University, the Wellcome Sanger Institute, and the Great North Children’s Hospital have identified a single gene mutation common to 11 pediatric patients with ...

Please provide your email address to receive an email when new articles are posted on . Infants with Artemis-deficient severe combined immunodeficiency improved after ...

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 ...

AGC Biologics Partners with Rarity PBC to Advance Life-Saving Gene Therapy for "Bubble Baby Disease" Collaboration will leverage AGC Biologics’ commercial manufacturing experience and proven platforms ...

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, ...

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 ...