News

Jasper Therapeutics, Avrobio Team Up to Assess JSP191

Jasper Therapeutics and Avrobio have entered into a non-exclusive research collaboration to evaluate the use of JSP191 in patients with Fabry disease who are being treated with one of Avrobio’s investigational gene therapies. Under this collaboration, JSP191 — an anti-CD117 monoclonal antibody — also will be…

Lucerastat Fails to Lessen Neuropathic Pain in Phase 3 Trial

Lucerastat, an investigational substrate reduction therapy for Fabry disease, has failed to outperform placebo at reducing neuropathic pain in patients, updated results from the MODIFY Phase 3 trial show. The findings mean that Idorsia‘s MODIFY study (NCT03425539) did not meet its primary goal, despite lucerastat being well…

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…