News

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…

Infection with COVID-19 may appear different and take a distinct clinical course in people with Cushing’s syndrome than in other patients, according to an international team of researchers. Despite an absence of data about the effects of COVID-19 on this high-risk group, researchers from…

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.

Cushing’s syndrome treatment Isturisa (osilodrostat) now is commercially available in France. The announcement from Recordati Rare Diseases comes soon after the medication became fully available for sale and distribution to adults in the U.S. The company stated that the medication will be made available elsewhere in the European Union…

Isturisa (osilodrostat) oral treatment for Cushing’s disease patients is now fully available to be sold and distributed across the United States, according to its developer Recordati Rare Diseases. The move follows Isturisa’s recent approval by the U.S. Food and Drug Administration (FDA) for adult…

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…

Strongbridge Biopharma has finished enrolling patients in its Phase 3 LOGICS clinical trial assessing the safety and effectiveness of Recorlev (levoketoconazole) to treat endogenous Cushing’s syndrome, the company announced. “The completion of enrollment of the Phase 3 LOGICS trial for [Recorlev] represents a significant milestone for Strongbridge and…

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…