U.S. Health Secretary Robert F. Kennedy Jr. on Tuesday added two rare genetic disorders, Duchenne Muscular Dystrophy and ...

This year saw notable progress in head and neck cancers, Huntington's disease, personalized genetic therapy, and heart ...

Scientists at the Icahn School of Medicine at Mount Sinai have developed a novel artificial intelligence tool that not only ...

The treatment uses a modified, harmless virus to deliver healthy genes to a patient’s cells to treat genetic diseases. Read ...

The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...

Scientists discovered that certain gene changes allow cells to function even when frataxin, the protein lost in Friedreich’s ...

The donation from Don and Anne Edwards aims to speed research, treatment and diagnosis for children with rare and genetic ...

The nonprofit is funding research at two universities—Oregon Health & Science University and UMass Chan Medical School—on a ...

When you buy through links on our articles, Future and its syndication partners may earn a commission. Babies born with harlequin ichthyosis have very distinctive features, including platelike scales ...

Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or less than 1 in 2,000 in Europe. 1-2 While each disease is individually rare, collectively, they represent a ...

Lurie Children's Hospital officially announced an expansion of research with the $11 million from Don and Anne Edwards, who ...

A 10-month-old baby who sparked nationwide headlines after receiving a first-of-its kind gene-editing treatment was released ...