News

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 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…

A celebrity lineup is slated for an upcoming fundraiser by the Scleroderma Research Foundation (SRF) to support disease research. The Cool Comedy, Hot Cuisine event will be livestreamed on Sunday, Oct. 17, at 5 pm PDT or 8 pm EDT. Tickets, starting at $100, can be purchased…

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…

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.

Bionews Insights is launching a new survey, in collaboration with Scleroderma News, with the goal of understanding how different aspects of disease affect an individual’s quality of life. The survey, which is expected to take approximately 15 minutes to complete, will use the WHOQOL-100 questions, developed by the World…

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…

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…

Chemomab Therapeutics is teaming up with the University of Leeds to investigate the role of the protein CCL24 in scleroderma (SSc) — particularly how it contributes to blood vessel damage. Their findings may aid in the clinical development of CM-101, Chemomab’s first-in-class antibody designed to target and…

Having pulmonary hypertension shortens survival in people with scleroderma, also called systemic sclerosis (SSc), according to a Brazilian study that followed patients for up to three years and used a computer algorithm to analyze the results. Specifically, this lower survival rate in SSc patients with pulmonary hypertension was…