Actuate Therapeutics (ACTU) announced that the U.S. Food and Drug Administration, FDA, has granted rare pediatric disease designation to ...

Amgen (AMGN) announced the presentation of new data across its rare disease portfolio and pipeline at the annual American College of ...

As Rare Disease day 2020 approaches, we take a look at the biggest challenges facing orphan drug developers and ask whether the future is bright or bleak for these difficult conditions.

The University of Minnesota’s annual Rare Disease Day seminar this year, held at the end of February, explained how patients with rare diseases can utilize telehealth services nationally and ...

Columnist Kristin Neva sometimes get overwhelmed by her increasing caregiver responsibilities, but it helps her to know she's not alone.

The FDA has said it will create a rare disease innovation hub to help speed the development of new treatments and build connections between developers and the rare disease community. The hub will ...

Columnist Sam Kirton encourages the IPF community to get a jump on legislative advocacy before the new congressional term begins.

Savara Inc. (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, today announced that members of its management team will be attending the below listed ...

The underlying cause of death is disclosed almost a month after the beloved Dodgers pitcher was pronounced dead from a ...

Los Angeles Dodgers icon Fernando Valenzuela died mainly as a result of septic shock ... The document also said he may have ...

Marinus Pharmaceuticals sells a drug to reduce seizures in people with a rare genetic disorder. Recently, a study showed the ...

AstraZeneca also said on Tuesday, along with its partner Daiichi Sankyo, it has submitted a new biologics license application ...