A good Samaritan called an audible and gave an 8-year-old battling a rare chronic disease the opportunity of a lifetime.

Her mother, Kelsey, living in Espanola, about 40 minutes west of Sudbury, welcomes all the activity because Iris has a rare, ...

Sidra Medicine, a member of Qatar Foundation, has established a gene therapy centre to treat rare genetic diseases such as ...

One in every 10 people worldwide is impacted by a rare genetic disease but about 50% of them remain undiagnosed despite rapid ...

"I knew that I had to do something to save Frederico, but also the other estimated 15,000 children diagnosed with the disease around the world each year," he says. In 2021, Goldsztein launched The ...

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches -- gene therapy ...

News on rare diseases, including the development of new treatments, the challenges of diagnosis and treatment and the stories of patients and their families. The page features in-depth reporting ...

Health department to open rare disease clinic at Government Medical College Hospital, Kozhikode, following parents' request for local treatment.

Health Minister Veena George announces registry for rare diseases, free treatments, and new clinic in Kozhikode.

Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...

The announcement was made at Arab Health 2025 where Sidra Medicine will highlight its international patient service programmes ...

MMR vaccines are safe and effective, but fewer children are getting them.