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 therapies ... 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 was ... Read more

May 19, 2020May 19, 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. The ... Read more

May 15, 2020May 15, 2020

Vitamin B3 Boosts Muscle Health in Mitochondrial Myopathy, Study Suggests

Mitochondrial Myopathy News, News

Niacin, vitamin B3, can help to normalize muscle metabolism in people with mitochondrial myopathy, a study based on a small pilot trial in patients suggests. Titled, “Niacin Cures Systemic NAD+ ... Read more

May 12, 2020May 12, 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. Survey ... Read more

May 8, 2020May 8, 2020

Gene Therapy Trial for Mitochondrial Disease of Retina Found Safe, Can Continue

News

An independent Data Safety Monitoring Board (DSMB) has found GS030, GenSight Biologics’ candidate gene therapy for retinitis pigmentosa, safe and recommended the company’s PIONEER trial continue as planned. PIONEER (NCT03326336) 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 their ... Read more

April 24, 2020April 24, 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. Called ... Read more

April 22, 2020May 22, 2020

Bezafibrate Does Not Induce Mitochondria Formation, Small Study Reports

News

A cholesterol-lowering medication, bezafibrate, taken daily for 12 weeks did not induce the formation of new mitochondria in people with mitochondrial myopathy carrying a mutation in the MTTL1 gene, a small observational ... Read more

April 17, 2020April 17, 2020

GenSight Readying to Ask EMA to Approve Gene Therapy for LHON

Leber's hereditary optic neuropathy, News

GenSight Biologics announced it has finished a pre-submission meeting with the European Medicines Agency (EMA), a key step in filing a request for the approval of Lumevoq, its gene therapy for Leber ... Read more