For Americans living with rare diseases, the Rare Pediatric Disease Priority Review Voucher Program is a vital tool. Yet its reauthorization is at risk.

Queen’s University has launched a new online resource to empower unpaid carers supporting their loved ones with a rare disease.

For more than 30 million people in the United States, living with a rare disease means facing lifelong challenges. Their journey often begins with uncertainty: an average diagnostic odyssey of five or ...

It is probably safe to say that progressive supranuclear palsy is not a household name. Unlike Parkinson’s or Alzheimer’s, conditions that have become part of the common vocabulary, progressive ...

“Having a rare disease is not the end. It’s actually the beginning,” shares Vesna, 36, a wife, mother, sister, friend, rare disease activist and person living with Gaucher disease, a rare genetic ...

The MarketWatch News Department was not involved in the creation of this content. National Organization for Rare Disorders (NORD(R) ) Brings Together 100+ Wis. Families for a Day of Hope, Learning, ...

Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with ...

Past the red carpets and the flashing lights of the paparazzi, some celebrities live in a reality more complicated than what ...

National Organization for Rare Disorders (NORD®) Brings Together 100+ Wis. Families for a Day of Hope, Learning, and Community During National Family Caregivers Month MILWAUKEE, Nov. 6, 2025—As ...