Ultragenyx’s Rare Bootcamp gives families a roadmap to help drive research and develop new treatments for rare diseases.

Jeff and Deena Leider are pushing federal officials to expand access to a treatment for their two sons' rare disease.

Rare disease families are increasingly driving research efforts to find treatments for their children. Ultragenyx’s Rare ...

CLEVELAND, Ohio — Browns owners Dee and Jimmy Haslam are giving $12.5 million to University Hospitals and an affiliated ...

Addressing the gathering, Union Health Secretary Smt. Punya Salila Srivastava highlighted that India’s rare disease response has evolved significantly over the past decade.

Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...

With a rare disease that affects only about 150,000 people in the US; one that runs in families.

In PEOPLE's exclusive clip from ‘One Day in My Body,’ Portia Cina, 18, shares her experience living with ichthyosis with ...

Rare disease patients often face years of uncertainty before getting a diagnosis, but new AI systems like DeepRare are cutting that wait. By combining symptoms, genetic data, and medical literature ...

Rare growth disorders are a diverse group of uncommon conditions that affect an individual’s physical development. They may stem from genetic mutations, hormonal imbalances, or other complex factors.