In Washington, even widely hailed programs with bipartisan support — like the rare pediatric disease priority review voucher ...

Three-year-old Logan Reese was diagnosed with transverse myelitis, a rare neurological condition caused by inflammation of ...

A federal advisory committee that provides guidance on which rare diseases newborns should be screened for has been dissolved ...

Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by ...

These slow, agonizing deaths can be prevented—but our new science-averse health czar apparently doesn’t see that as a priority in his Make America Healthy Again crusade. Along with the 10,000 or so ...

At only two days old, Sophie was losing too much weight, and too quickly. Further genetic testing would show that Sophie has ...

The FDA has granted Rare Pediatric Disease Designation for NEO100 in the treatment of pediatric-type diffuse high-grade ...

Kolkata: A three-year-old girl waiting for treatment for a rare disease died without getting any medical assistance under the ...

Knight says that the method of transmission that Collinge identified is “extremely unusual,” and there’s no evidence that ...

With the annual cost estimated to be about ₹1 crore for each child requiring treatment for ultra-rare diseases, the Medical ...

The United States recorded three measles deaths in the first 24 years of this century. In just over three months of 2025, it ...

The Genetic Architecture of Congenital Diarrhea and Enteropathy - (https://www.nejm.org/doi/10.1056/NEJMoa2405333) ...