An experimental gene therapy developed to treat children born with a rare immunodeficiency disease has demonstrated extraordinary efficacy according to a new long-term follow-up study published in the ...

Doctors have seemingly found a way to cure SCID in babies: by inserting a corrective gene into a patient's cells via a tweaked version of HIV A revolutionary new study has shown that the human ...

A long-term follow-up study tracking a group of children who received experimental gene therapy as part of an early-stage clinical trial is reporting the treatment is safe and effective up to 10 years ...

Screening newborns for severe combined immunodeficiency disease (SCID) significantly increases the survival of children after bone marrow transplantation, a new North American study finds. Published ...

Severe Combined Immunodeficiency (SCID) is a rare, life-threatening immunological disorder characterized due to the lack of B-lymphocyte and T-lymphocyte functioning. Sporadic (occasional) ...

Severe combined immunodeficiencies (SCIDs) comprise a group of rare, monogenic diseases that are characterized by an early onset and a profound block in the development of T lymphocytes. Given that ...

The study population consisted of patients with documented Peyronie's disease and penile curvature of at least 1-y duration who underwent either surgical correction of their penile curvature or ...

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, ...

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 ...

Researchers used an experimental gene therapy to develop a possible treatment for a rare and deadly immune disorder known as “bubble boy” disease, the National Institutes of Health (NIH) announced ...

A new immunodeficiency disease caused by a novel genetic mutation has been identified by researchers, providing unique insights into cell biology. A new immunodeficiency disease caused by a novel ...

A new article is aimed at pediatricians, family physicians and other doctors who may treat newborns, including those who appear healthy at birth but begin to get severe, repeated infections requiring ...