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

Actio Biosciences, a clinical-stage biotechnology company advancing the translation of genetic insights into novel precision ...

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.

The seven-year medical odyssey that once defined rare disease diagnosis is being rewritten by algorithms capable of identifying medical zebras in the time it takes to refresh your social media feed.

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 ...

The Food and Drug Administration accepted Opus Genetics's investigational gene therapy into its Rare Disease Evidence Principles program, the company said Monday. The clinical-stage biopharmaceutical ...

Across America, millions of parents of children with rare diseases are in a race against time, hoping that new treatments will be developed fast enough to save their kids. Thanks to advances in ...

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 ...

The Boards of Directors of Angelini Pharma and Catalyst Pharmaceuticals have unanimously approved the acquisition of Catalyst Pharmaceuticals at ...

Cleveland Browns owners recently made a large donation for cancer research. Dee Haslam lives daily with a rare blood cancer ...

Hannah Strickland is only 9 years old, but her big personality is capturing hearts online. Whether she's describing her ramen ...