Our goals are to expand access to genomic testing and develop cutting-edge genomic testing to help more families get answers ...

Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, ...

Rare eye diseases are the leading cause of untreatable blindness in Europe and affect people of all ages. The RESTORE VISION ...

And today we have two segments on rare diseases. Later, we'll hear from NFL legend, Boomer Esiason. But first, I want to welcome Dr. Marshall Summar, CEO of Uncommon Cures, and Pamela Gavin, CEO of ...

For the first time, Hospital Kuala Lumpur (HKL) is proudly hosting the annual Rare Disease Day 2025 celebration to raise ...

ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, ...

Local nonprofit Jack's Generosity hosted an event called Unique Jeans to raise awareness for rare ARS gene diseases.

Donna Reynolds of Richmond knows all too well the devastating toll that a rare disease can take, not just on an individual, but on an entire family.

Zevra used the awareness day to roll out the “Learn NPC, Read Between the Signs” disease awareness campaign. The biotech won ...

Williams, who has performed the National Anthem at baseball stadiums as part of Boehringer Ingelheim’s Breathless campaign, ...

Rare Disease Day, observed on Feb. 28, brings attention to uncommon diseases affecting populations of 200,000 or fewer. In ...

MADISON, Wis. (WIFR) - For 14 years, Treyson Wallace’s family searched for answers. Born in 2009, Treyson failed a newborn ...