The Food and Drug Administration is reconsidering its decision not to approve a drug that an Omaha mom says is saving her son ...

Researchers led by UC Davis Health scientist Sanchita Bhatnagar have developed a promising gene therapy that could treat Rett ...

Researchers led by UC Davis Health scientist Sanchita Bhatnagar have developed a promising gene therapy that could treat Rett syndrome. The therapy works on reactivating healthy but silent genes ...

Severe iron deficiency can lead to pica, a condition where you want to eat things with no nutritional value. Both Peterson ...

Researchers at Tohoku University have discovered that an oral drug called MA-5 can improve both heart and muscle problems in Barth syndrome, a rare genetic disorder affecting 1 in 300,000 births ...

Researchers at Tohoku University have discovered that an oral drug called MA-5 can improve both heart and muscle problems in Barth syndrome, a rare genetic disorder affecting 1 in 300,000 births ...

Barth syndrome is a rare genetic disorder with no known cure. Researchers at Tohoku University examined a new oral drug called MA-5 that could provide life-changing relief to these young patients.

SAN FRANCISCO (KPIX) -- With the FDA recently rejecting a drug application that aims to help those living with Barth Syndrome, one Bay Area family is frustrated by the delay of approved treatments ...

Approximately 150 people live with Barth Syndrome nationwide, an ultra-rare genetic disorder that affects only males and impacts muscle and heart muscle health.