GeneDx, a fast-growing biotech doing exome and genomic testing, has seen big returns on Wall Street as appetite for its tests ...

The programme will focus on accelerating treatments for rare diseases, most of which currently lack approved therapies.

The Senate failed to pass a massive spending bill on Thursday—which includes the rare pediatric PRV program but also funding ...

Actor turned rare disease advocate Luke Rosen tells how his daughter, Susannah, lives with a KIF1A-associated neurological ...

The drug, which has suffered multiple setbacks over the years, could finally reach the market as a treatment for a form of ...

Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis ...

Hackathons using AlphaGenome and other AI models are hunting down the genetic causes of devastating conditions that have ...

Research on rare diagnoses and the development of precision medicine depend on patients being able to share their health data in a secure and ethical manner. The research study, published in ...

Oxana Iliach is a Senior Director of Regulatory Strategy at Certara (PA, USA), a biotech company that offers comprehensive solutions for transforming and accelerating drug development. In her spare ...

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 ...

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 ...