Dr Sudheendra Rao N R from the Organization for Rare Diseases India reveals that approximately 70 million are affected by ...

The development of AMX0318 utilized Gubra’s proprietary streaMLine platform, which is an AI-driven technology used to ...

The Priority Review Voucher (PRV) program is designed to incentivize rare disease drug development.

What is eosinophilic fasciitis, and how is it diagnosed and treated? Learn about this rare fascia condition in simple terms.

After years stuck in the “doldrums,” the biopharma sector is in a “very good place” heading into the new year, analysts told ...

No standard treatments for the sometimes deadly disorder have been developed, but that may change due to the newly published ...

Globally, there are more than 300 million people who have one of the 7,000 known rare diseases (Nguengang Wakap et al., 2020). In the United States, any disease affecting fewer than 200,000 people per ...

DANBURY, Conn., Aug. 25, 2025 /PRNewswire/ -- The National Organization for Rare Disorders (NORD®), the leading national nonprofit serving more than 30 million Americans with rare diseases, today ...

QUINCY, Mass., Aug. 13, 2025 /PRNewswire/ -- The National Organization for Rare Disorders (NORD®), a leading national nonprofit serving more than 30 million Americans with rare diseases, announced ...

Now 4, Cade Jobsis has been receiving treatment that helps keep him strong. But with funding pulled, his parents don't know how much longer he'll get it Leah Serena Photography After going through the ...