News

The U.S. Food and Drug Administration has granted orphan drug designation to Theraly Fibrosis’ TLY012 for the treatment of systemic scleroderma. This designation is given to medications with the potential to treat rare conditions — those that affect fewer than 200,000 people in the U.S. Benefits include…

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…

With the World Scleroderma Foundation (WSF)’s support, experts worldwide answered questions in the form of  recommendations for scleroderma patients amid the COVID-19 outbreak, including the importance of maintaining current medications and discussing any changes with their doctors. Their guidelines, “Systemic sclerosis and the COVID-19 pandemic:…

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…