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

Families, campaigners and clinicians are calling for more public money to be provided to pay for access to drugs which could ...

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

Rare diseases, defined as conditions that affect a small percentage of the population, represent a significant challenge in medical research and healthcare. Collectively, these diseases encompass a ...

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

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 ...

When our son Peter was diagnosed with an ultra-rare form of muscular dystrophy at the age of 10, the first question we asked was: Is there a treatment? The answer was no. By the time our daughter ...

Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or less than 1 in 2,000 in Europe. 1-2 While each disease is individually rare, collectively, they represent a ...

Market exclusivity, fast-track approvals and high pricing have led to a slew of PE investments in pharma companies specializing in treatments for rare diseases. Often called orphan drugs, the costs of ...

As Ekterly nears the one-year anniversary of its FDA approval, Italy’s Chiesi Group is making moves to acquire the rare ...