Michael Hund, Rare Ventures founder and CEO and EB Research Partnership CEO, joins 'Squawk Box' to discuss funding the next ...

BioMarin will acquire Alesta Therapeutics for $275 million upfront, bolstering BioMarin's unit focused on rare skeletal ...

Boehringer Ingelheim has continued its rise up patient groups’ ranking of rare disease companies, adding to last year’s gains to cement its status toward the top of the leaderboards. | Boehringer ...

Nome uses AI and drug-development expertise to help rare disease families turn genetic diagnoses into potential paths toward ...

By combining long reads, genome assembly, diverse reference data, and AI, researchers aim to find rare disease variants that ...

Advocates and politicians are urging Government and the HSE to reimburse the cost of Friedreich’s ataxia drug Skyclarys ...

Regeneron’s Pasatru is the second fibrodysplasia ossificans progressiva drug approved in the U.S., following the 2023 ...

A new Pittsburgh-based initiative, Rare Ventures, has launched with a $25 million commitment from the Richard King Mellon Foundation to accelerate rare disease treatment development. Led by the EB ...

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.

Using the US Food and Drug Administration’s (FDA) plausible mechanism framework to allow for greater use of causal inference ...

Dermatology drugmaker Leo Pharma and the scattering of patients across the globe with rare skin disease generalized pustular psoriasis (GPP) are circling Aug. 13 on their calendars as the second ...

When his infant son was diagnosed with a rare disease, a Canadian father was dismayed to discover there was no treatment or cure. So he set out to make one himself. Terry Pirovolakis, an IT director ...