Researchers led by the University of Vienna and Liège University Hospital Center have identified genetic variants associated ...

The Digital Health Measurement Collaborative Community (DATAcc) by the Digital Medicine Society (DiMe) today announced the ...

Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging ...

Rare diseases affect fewer than 200,000 people in the U.S., approximately 30 million individuals. Sadly, 3 out of 10 children with a rare disease won’t live to see their fifth birthday, yet the path ...

Poster presentations at ASH highlight advances in the understanding and research of Castleman disease, cold agglutinin disease, B-cell lymphoma, and other hematologic conditions "We are particularly ...

Rare disease clinical trials are smaller and often use surrogate endpoints, reducing costs but posing unique operational ...

The FDA has outlined a new review process for drugs and biologics designed to treat ultrarare genetic diseases that would allow a single-arm trial, plus other supportive data, to serve as pivotal ...

For patients facing a rare disease, a genetic test isn’t the end of their story; it’s often just the beginning. A test result may point to a diagnosis, or it may reveal a genetic variant that we have ...

Results from CADENCE, the first and largest multinational prospective registry for patients with CAD/CAS, describe safety events observed in patients treated with sutimlimab, with an average treatment ...

The MarketWatch News Department was not involved in the creation of this content. Results from CADENCE, the first and largest multinational prospective registry for patients with CAD/CAS, describe ...