Shane DiGiovanna was born with epidermolysis bullosa, or EB, a condition he said is described as "the worst disease you’ve ...

The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient ...

The FDA has approved a generic drug for a very rare brain disorder, while walking back suggestions by Trump it showed promise for people with autism. (AP Photo) ...

A pivotal medical milestone has been reached. For the first time ever, researchers have used a personalized CRISPR-based gene therapy to treat an infant’s rare and life-threatening illness. Doctors at ...

Add Yahoo as a preferred source to see more of our stories on Google. This photo provided by Emily’s Entourage in April 2025 shows Emily Kramer-Golinkoff, who has cystic fibrosis caused by a rare ...

Electrical stimulation of the spinal cord strengthened the muscles of three people with spinal muscular atrophy, a rare motor neuron disease. An experimental treatment that stimulates the spinal cord ...

In September, Trump and the FDA commissioner announced the drug was under review to benefit patients with autism. The FDA didn't find evidence of that.

But FDA officials said Monday that the agency later focused only on the strongest scientific evidence. That evidence only supported the drug’s use for people with the rare genet ...

Marley Mansour, 11, is tickled by mom Kayla Mansour on her bed at their home in Rowlett on Tuesday, April 29, 2025. Marley is set to make medical history as the first person in the world to receive ...

At 18 months old, baby Luca has endured more than most people will in their lifetime. Australian parents Ashleigh and Cody welcomed their little boy at just 33 weeks – six weeks earlier than expected ...

Psychiatric disorders can share common genetic influences, which means parts of DNA can be at the root of more than one mental condition, new research has found. The study, led by researchers at Texas ...

An international collective of researchers is delivering new insights into why having multiple psychiatric disorders is the norm rather than the exception. In a study published today in the journal ...