News

People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…

Treatment with Takhzyro (lanadelumab) effectively reduces the number of attacks in patients with hereditary angioedema even in early phases of treatment, new analysis of data from the Phase 3 HELP study shows. Most recent trial results were shared in a poster, “Lanadelumab demonstrates rapid and sustained prevention of hereditary…

The unique mode each specific compound binds to and blocks the enzyme plasma kallikrein could lead to the development of improved therapies for patients with hereditary angioedema (HAE), according to a new study. The research, “Structures of full-length plasma kallikrein bound to highly specific inhibitors describe…

KalVista Pharmaceuticals‘ investigational therapy KVD900 for the treatment of hereditary angioedema (HAE) rapidly reduces the activity of plasma kallikrein, one of the drivers of HAE attacks. That promising result comes from the first-in-human Phase 1 clinical trial of KVD900, and was shared at the 11th C1 Inhibitor Deficiency…

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…

Daily treatment with BCX7353, an investigative oral inhibitor of plasma kallikrein by BioCryst Pharmaceuticals, was well-tolerated and lowered attack rates in hereditary angioedema (HAE) patients included in a Phase 3 trial. Both 110 mg and 150 mg doses provided a significant benefit by decreasing HAE attacks — the APeX-2 trial’s primary…

Prophylactic (preventative) treatment with Cinryze reduced the number and severity of attacks in young children with hereditary angioedema (HAE), without significant adverse events, a Phase 3 trial shows. The study, “A Randomized Trial of human C1 inhibitor prophylaxis in children with hereditary angioedema,” was published in Pediatric…

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies (NCATS) at…

With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…

Takhzyro (lanadelumb), an antibody already approved for routine prevention of hereditary angioedema (HAE) attacks in patients ages 12 and older, was found to safely reduce the number of monthly attacks experienced by adolescents in a Phase 3 clinical trial. The study, “Efficacy and safety of lanadelumab for prophylactic treatment…