News

May 29, 2020May 29, 2020

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

News, syndicated

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 ... Read more

May 25, 2020May 25, 2020

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

News, syndicated

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 ... Read more

May 20, 2020

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

News, syndicated

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. ... Read more

May 15, 2020May 15, 2020

Enrollment Completed for Phase 3 Trial Testing Recorlev for Endogenous Cushing’s Syndrome

News

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 ... Read more

May 13, 2020May 13, 2020

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

News, syndicated

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. ... Read more

May 8, 2020May 8, 2020

Pandemic Delays Trials Investigating Relacorilant as Endogenous Cushing’s Syndrome Treatment

News

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 ... Read more

April 29, 2020April 29, 2020

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

News, syndicated

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 ... Read more

April 24, 2020April 24, 2020

Isturisa for Cushing’s Syndrome Likely To Be Available in Europe Over Summer

News

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 ... Read more

April 22, 2020April 22, 2020

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

News, syndicated

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. ... Read more

April 17, 2020April 17, 2020

Pituitary Adenomas in Cushing’s Disease Differ Between Men, Women, Study Reports

News

The pituitary adenomas that cause Cushing’s disease in men differ from those in women in hormone content and gene expression patterns, a study shows. These findings suggest that biological ... Read more

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