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…
The Phase 3 GRACE clinical trial evaluating Corcept Therapeutics‘ relacorilant as a potential treatment for high blood pressure and poor glucose tolerance in people with endogenous Cushing’s syndrome is expected to take longer to complete, the company announced. While patients in the GRACE study continue to…
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…
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…
Recordati Rare Diseases announced that it has officially acquired marketing rights in Europe for the Cushing’s treatment Isturisa (osilodrostat), enabling the company to distribute it throughout the European Union, as well as in the U.K., Norway, Liechtenstein, and Iceland. The company — a subsidiary of Recordati, based in Italy…
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