After five years, Nido Biosciences has reached the end of the road, closing its doors after its lead neurological disease ...

A rare form of diabetes is emerging in the very first weeks of life, reshaping how doctors think about blood sugar disorders ...

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.

Foresee Pharmaceuticals has shown that a long-acting version of its injectable prostate cancer med Camcevi can help patients ...

More than 500 European patients with previously undiagnosed rare disorders received a genetic diagnosis through research carried out by an extensive European collaboration, the Solve-Rare Diseases ...

ANI Pharmaceuticals’ ANIP rare disease franchise spearheaded its growth in 2025. This robust growth was fueled by the strong ...

BioMarin is acquiring Amicus Therapeutics for $4.8 billion, merging two rare disease-focused biotechs founded around the turn ...

International Angelman Day also marks the birthday of Dr. Harry Angelman, the British pediatrician who first identified the ...

Inspired by his father, Grandview High School student Josue Clara started a nonprofit called Beyond Rare to build awareness ...

The National Institutes of Health established the Genomics Research to Elucidate the Genetics of Rare Diseases (GREGoR) Consortium in 2021 with the goal of finding molecular diagnoses for individuals ...

Source: Mark L. Ruffalo, D.Psa., L.C.S.W. Psychosis has historically been defined as "the loss of contact with reality" and can be observed in various mental disorders, including schizophrenia, ...