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Scientist diagnosed with the rare disease that took her mother's life dedicates her life to finding a cure
Sonia Vallabh had already obtained her law degree from Harvard, but after her diagnosis, decided to retrain in biomedical ...
Inspired by his father, Grandview High School student Josue Clara started a nonprofit called Beyond Rare to build awareness ...
For a technology that costs millions, a collaborative approach may improve efficiency and allow the science to live up to its ...
Ipsen’s oral small molecule has failed to reduce the progression of a genetic disease that gradually turns tendons and ...
BioMarin is acquiring Amicus Therapeutics for $4.8 billion, merging two rare disease-focused biotechs founded around the turn ...
Cure Rare Disease (CRD) announced it has been awarded a $7.4 million grant from the California Institute for Regenerative ...
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 ...
A girl diagnosed with a rare brain disease after suddenly becoming unresponsive at home has been "fighting like hell", her ...
A new monoclonal antibody called nebokitug offers fresh hope to patients who have primary sclerosing cholangitis (PSC), ...
BioMarin Pharmaceutical said on Friday it would acquire Amicus Therapeutics for about $4.8 billion, in the drugmaker's second ...
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 ...
Danish pharmaceutical multinational Novo Nordisk is expanding its presence in inherited blood disorder treatments ...
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