A provision protecting expensive medications, used to treat multiple rare diseases, from the Medicare drug price negotiations ...

Patients with rare diseases are increasingly working to find and fund their own cures. But should they have to?

With CT scans picking up more cases of sclerosing mesenteritis, be prepared to diagnose this poorly understood autoimmune ...

Expansion of the Medicare drug price negotiation rare disease drug carve-out would unnecessarily limit the number of drugs ...

For Bethany Duckworth, being an active mom wasn’t just a dream, it was her plan. "It's depressing," Duckworth said. "It's sad ...

June is Alzheimer's and Brain Awareness Month. Colbie Schueneman, 6, is sharing her story with KIF1A, also known as KAND.

When nerve cells don’t end up in the right place during brain development, it can cause periventricular heterotopia —a ...

Here is basic, but essential information on what you should know about Powassan, a rare and deadly disease spread by ticks.

Harlequin ichthyosis affects just a handful of newborns each year in the US, but the condition is unmistakable, marked by a ...

The Mighty Maverick Muscle Run was held at the Miller Ecological Park in Lebanon in support of local 3-year-old born with a ...

With the FDA recently rejecting a drug application that aims to help those living with Barth Syndrome, one Bay Area family is ...

In May 2025, researchers announced that K.J. Muldoon, a baby boy born without the ability to process dietary protein properly ...