Astria Therapeutics is gearing up to request permission for clinical testing in the U.S. of STAR-0215, its experimental therapy to prevent swelling attacks in hereditary angioedema (HAE). The company is expected to submit a formal request — in the form of an investigational new drug application — to the…
About one year of treatment with Takhzyro (lanadelumab) safely and effectively lowered the frequency of swelling attacks, while improving quality of life and treatment satisfaction in people with hereditary angioedema (HAE), according to interim data from the real-world EMPOWER study. In addition, nearly 1 in 5 patients was able to…
KalVista Pharmaceuticals has launched a Phase 3 trial, called KONFIDENT, to test the safety and effectiveness of KVD900, an experimental oral and on-demand treatment for swelling attacks in people with hereditary angioedema (HAE). “Beginning the KONFIDENT trial represents a major milestone for KalVista,” Andrew Crockett, the company’s…
Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…
Long-term treatment with Orladeyo (berotralstat) at its approved 150 mg daily dose reduces the rate of swelling attacks significantly and improves quality of life for people with hereditary angioedema (HAE), according to nearly two years of data from the Phase 3 APeX-2 trial. These benefits were observed to…
Newbury Pharmaceuticals is launching its injectable therapy of icatibant, the active agent of Firazyr, in Sweden and Norway to treat acute attacks in people with hereditary angioedema (HAE). “Making Icatibant Newbury available in Sweden and Norway is an important milestone to Newbury as we see the fruits…
Bionews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A Window…
A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that burden,…
CSL Plasma has donated $125,000 to the American Red Cross to support recovery and relief efforts following disasters in the U.S., including tornadoes, hurricanes, wildfires, residential fires, and floods. These disasters can affect areas where plasma donors live and where employees involved in the manufacturing of plasma-derived therapies…
The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…
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