The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…
Treatment with lenabasum is safe and leads to clinically meaningful improvements in people with diffuse cutaneous scleroderma (dcSSc), results from Phase 2 trial show. The therapy is now being tested in this trial’s extension study, as well as in a Phase 3 trial in adults with dcSSc.
A triple combination of prednisolone, cyclophosphamide and plasma exchange (plasmapheresis) was effective against skin thickening in seven of eight people with diffuse cutaneous systemic sclerosis (dcSSc), a case series study reports. These results support further investigation into treating newly diagnosed dcSSc patients with this combination…
FFF Enterprises and Bionews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone. Recognized as the nation’s leading supplier of…
While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…
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…
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