Tag Archive for: rare disease

The agency’s top five represent an eclectic mix of cancer, cardiovascular and rare disease drugs.

The company delayed the timeline for reporting key data from a trial of its cannabis-based drug to treat a rare genetic disorder, citing difficulties in enrolling patients amid a rise in respiratory illnesses.

If approved, the treatment, branded Hemgenix, will be the first gene therapy in the European Union for the condition that is usually treated by regular injections of factor IX, the European Medicines Agency said.

The companies have initiated a research agreement supporting the development of new AI-ECG algorithm to help identify patients who may be at risk of cardiac amyloidosis.

To help marginalized patients feel comfortable with their care, healthcare communicators must expand our library of stories.

Amgen confirmed Monday it will acquire rare disease giant Horizon Therapeutics for $26.4 billion.

The Ireland-based company issued a statement confirming that it is engaged in “highly preliminary discussions” with Amgen, Janssen, and Sanofi. Any acquisition of Horizon Therapeutics would be among the biggest deals of 2022.

The regulator, known as CNMC, said the pharmaceutical company had abused its dominant position as holder of the only medicine available in Spain against the rare genetic disease Cerebrotendinous Xanthomatosis.

Mereo BioPharma will cut 40% of its headcount and significantly reduce expenses to support the advancements of its lead programs through the clinic, the company announced Tuesday. 

Horizon Therapeutics achieved third place in the 2022 OCI top companies ranking namely for its “Gout Revisited” campaign, which seeks to remove the stigma of gout, a disease that has been largely coined as an affliction of the “overindulgent, rich white man.” Keli Walbert, executive vice president, U.S. Operations, at Horizon Therapeutics shared insights with MedAdNews about the company’s strategy in debunking gout myths and steering the message in the right direction.