News

Ofev Seen to Slow Lung Function Decline Across SSc-ILD Subgroups

Ofev (nintedanib) effectively slowed lung function decline in adults with interstitial lung disease (ILD) associated with systemic sclerosis (SSc-ILD) across patient groups based on autoantibody status and skin involvement severity, according to an analysis of the SENSCIS trial. These findings add to the trial’s top-line results,…

FDA Grants Orphan Drug Status to Cudetaxestat

Cudetaxestat, a potential anti-scarring medication, has been granted orphan drug status by the U.S. Food and Drug Administration (FDA) for the treatment of scleroderma. “There are limited options and high medical need for patients suffering from systemic sclerosis [SSc, or scleroderma],” Daven Mody, vice president of regulatory affairs…

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…

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…

‘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.

Surveyed Patients Report Satisfaction With Treatment

Most scleroderma patients who responded to a U.S.-based online survey report being satisfied with their current medications and are not considering switching treatments. However, only about half of survey respondents reported being satisfied with their overall life quality, or optimistic about the future, perhaps highlighting gaps in care for…

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

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…