My 2-year-old's condition was extraordinarily rare, affecting fewer than two children in a million, doctors told us.

Our inbox has been flooded with emails from viewers and readers who told us how their lives have been affected by rare diseases.

Did you know neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) is a rare genetic disorder that can cause tumors ...

From February 24-26, 2026, the EveryLife Foundation for Rare Diseases (EveryLife Foundation) convened hundreds of patients, caregivers, advocates, researchers, and policymakers from 49 states, the ...

St. Louis Families Unite for Rare Disease Day 2026 as Take Part Foundation Advances Critical Genetic Testing and ...

The U.S. Senate unanimously approved a bipartisan resolution declaring Feb. 28, 2026, as Rare Disease Day, aiming to expand ...

As Malaysia commemorates National Rare Disease Day and marks the launch of the National Policy for Rare Diseases, long-term, ...

AYVAKIT ® (avapritinib) is a prescription medicine used to treat adults with indolent systemic mastocytosis (ISM). AYVAKIT is not recommended in people with low platelet counts (less than 50 x 10 9 /L ...

While the vast majority of rare diseases lack approved, specific treatments or cures, health experts emphasize the importance of regular consultations and treatments that not only manage symptoms but ...

Living with Isaacs' syndrome, a rare neurological condition, has made experience with healthcare challenging. Despite millions affected, support is limited.

The Caribbean Community (CARICOM) grouping is calling for stronger surveillance system amid on going efforts to deal with the ...

Discover incredible stories of celebrities like Selena Gomez and Lady Gaga facing rare diseases. See how these captivating talents thrive while living with rare health conditions.