News

RARE-X, Global Genes to Help Collect Rare Disease Patient Data

In their continued efforts to improve health equity for people with rare diseases, Global Genes and RARE-X have joined forces to help advocacy groups collect patient data and make the most of that information. “Patient data is perhaps the most valuable asset rare disease communities can leverage to…

Patients Say They Want to Switch to Less Frequent Therapies

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, Diversity Coalition Team Up to Advance Health Equity

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…

HAEi Offers New Advocacy Courses, Patient App in 14 Languages

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.

Rare Disease Diversity Coalition Awards $600K to Combat Disparities

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…

Fewer HAE Attacks Evident With Takhzyro Use in Canadian Study

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…

‘Rare’ Documentary in Kickstarter Campaign to Raise $45K by Oct. 28

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.