News

The European Commission has approved Ofev (nintedanib) to preserve lung function in adults with systemic sclerosis-associated interstitial lung disease (SSc-ILD). This decision follows the recent recommendation from the Committee for Medicinal Products for Human Use, a branch of the European Medicines…

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…

Blocking the CCR2 protein reduced dermal tightening, lung fibrosis (scarring), and the infiltration of immune cells in the skin in a mouse model of scleroderma, a study shows. The study, “Antifibrogenic effects of C‐C chemokine receptor type 2 antagonist in a bleomycin‐induced scleroderma model,” was…

Horizon Therapeutics announced it plans to pursue the development of HZN-825, a potential therapy for diffuse cutaneous systemic sclerosis (dcSSc) that it gained in acquiring Curzion Pharmaceuticals. Horizon plans to conduct a study this year to investigate the pharmacological profile of HZN-825, previously known as CZN001. Company leaders…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

Emerald Health Pharmaceuticals has launched a clinical trial evaluating EHP-101, its investigational cannabidiol-derived therapy, in adults with diffuse cutaneous systemic sclerosis (dcSSc). “This is our first Phase 2 clinical study, which is another important milestone for our lead product candidate,” Joachim Schupp, MD, Emerald Health’s chief…

The active ingredient in EHP-101 — known as VCE-004.8, derived from synthetic cannabidiol — is not classified as a controlled substance under Canada’s Controlled Drug and Substance Act (CDSA),  the potential treatment’s developer, Emerald Health Pharmaceuticals, reported. VCE-004.8 is a non-reactive, non-psychotropic synthetic compound derived from cannabidiol, or CBD,…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

Levels of a subtype of immune B-cells in the blood are increased in some people with scleroderma and associated with lower respiratory function, higher blood pressure in the lungs, and more kidney damage, a study reports. The study, “CD21-low B cells in systemic…