According to the open letter, the delay in rolling out the proposed ₹ 974 crore national programme on rare diseases has exacerbated these challenges, leaving patients, most of whom are children, in ...

Patient advocacy groups warned that delays in implementing India's rare disease policy have left many, including children, in ...

Hirschsprung disease is a rare condition affecting one in every 5,000 babies, according to the National Institutes of Health.

Robert F. Kennedy Jr. has spasmodic dysphonia, a voice disorder caused by a malfunction in the brain. Here’s what to know.

The only drug that treats children with a rare degenerative disease called Batten disease could be withdrawn within the next ...

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.

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

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

At just 19, Geetika Iyer made a life-changing decision to donate a part of her liver to save her father, whose health was ...

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