News

Patients with hereditary angioedema (HAE) are willing to try new therapies that can effectively lessen disease burden with less frequent dosing, according to an interview-based study conducted by Astria Therapeutics. The findings were shared in a poster, titled “Burdens of Disease and Treatment in Hereditary…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

HAE International (HAEi) has expanded its free online training platform to include new courses on advocacy, disease-related information, and resources. “The HAEi Advocacy Academy is the premier online learning platform for the HAE Community,” Fiona Wardman, the non-profit’s chief regional patient advocate, said in a press release.

Adalvo has completed a mandatory requirement for a marketing authorization in the European Union for its generic version of Firazyr (icatibant injection), which is approved for acute attacks in adults with hereditary angioedema (HAE), the company has announced. The successful closure of the decentralized procedure came more…

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

Use of Takhzyro (lanadelumab) for at least six months led to fewer hereditary angioedema (HAE) attacks and treated attacks, a case review study from Canada reported. Three of its 12 patients went into complete remission, and all enjoyed a more comfortable social life, the investigators noted. The study, by…

The New Zealand Medicines and Medical Devices Safety Authority has authorized Intellia Therapeutics to launch a Phase 1/2 trial testing NTLA-2002, its investigational gene-editing therapy, in adults with hereditary angioedema (HAE). According to the company, patient enrollment is expected to start by the end of the year. Data…

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.

CAMP4 Therapeutics’ platform that targets a class of RNA known as regulatory RNAs, or regRNAs, can improve the activity of the gene that is defective in most cases of hereditary angioedema (HAE). That is according to new preclinical data shared by scientists from CAMP4 at the 17th Annual…

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…