News

NORD Fundraiser Sets 7,000-Mile ‘Bike, Run or Walk’ Goal for 7,000 Rare Diseases

This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least 30 million Americans. The campaign kicks off Feb. 1 and culminates with Rare Disease Day on Feb. 28. Organized by…

Phase 1 Trial of Oral Therapy KVD900 for Hereditary Angioedema Underway

An oral candidate for the treatment of hereditary angioedema (HAE) has begun a phase 1 clinical trial, KalVista Pharmaceuticals recently announced. HAE is a rare genetic disease characterized by recurrent episodes of severe swelling (angioedema) and potential pain, nausea, and vomiting. It occurs in about 1 in 10,000 to 1…

FDA to Review Pharming’s Application for Ruconest as HAE Swelling Prevention

The U.S. Food and Drug Administration (FDA) has agreed to review Pharming’s application to get Ruconest (conestat alfa) approved for the routine prevention of swelling attacks caused by hereditary angioedema (HAE). The therapy already is approved for the acute treatment of swelling attacks in adults and adolescents with…