An Italian foundation that helped a young boy overcome Wiskott-Aldrich syndrome becomes the first non-profit in the world to ...

Researchers developed a new gene-editing strategy that dramatically boosts the effectiveness of gene therapies in the liver, a breakthrough that could lead to new treatments for about 700 genetic ...

On this episode, an infant with a harrowing diagnosis defies all odds after receiving a $2 million genetic treatment. Them, a ...

The nonprofit foundation, n-Lorem developed personalized medicine for a child with rare leukodystrophy.

Children with avoidant restrictive food intake disorder (ARFID) have an elevated risk of developing psychiatric and physical ...

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

Poor regulation and lack of transparency in Africa's fertility industry leave sperm donors and recipients vulnerable to ...

Joe Lang has helped shape California policy as a lobbyist since the 1980s. For the past decade, he's pushed to find a cure ...

Shane Burcaw has a genetic disease and uses a wheelchair, his wife Hannah Burcaw does not. On their YouTube channel, they ...

A new study from Lund University in Sweden suggests that young children who eat more fiber may have a lower risk of ...

The National Organization for Rare Disorders is a coalition of patient advocacy groups and the first national nonprofit that ...

A Walsall charity which delivers vital help to those suffering with sickle cell disease is celebrating its fifth anniversary ...