News

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…

Enrollment is open for the open-label, Phase 1/2 HAERMONY trial to evaluate the safety and effectiveness of BMN 331, an investigational gene therapy for hereditary angioedema (HAE). The trial’s sponsor, BioMarin Pharmaceutical, plans to recruit about 34 adults with type 1 or type 2 HAE at one site…

It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.

Orladeyo (berotralstat), an approved therapy for preventing swelling attacks in hereditary angiodema (HAE), was effective at controlling attacks in an HAE patient with normal C1 inhibitor activity, a case study reported. The clinician said this oral therapy may be particularly suitable for patients who have a needle phobia…

The U.S. Food and Drug Administration (FDA) has approved the use of a prefilled syringe of Takeda’s Takhzyro (lanadelumab) to prevent swelling attacks in hereditary angioedema (HAE) patients, 12 and older. The prefilled syringe comes fully assembled and requires less preparation than the current vial-based injection of…

Short-term treatment with Berinert, a plasma-derived form of the C1 inhibitor protein, successfully prevented acute swelling attacks in a patient with hereditary angioedema (HAE) following oral surgery, a case report describes. The report, “Oral Surgery Procedures in a Patient Affected by Hereditary Angioedema Type I,” was published…

Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…