Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from ... Read more
The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting ... Read more
An independent Data Safety Monitoring Board (DSMB) has found GS030, GenSight Biologics’ candidate gene therapy for retinitis pigmentosa, safe and recommended the ... Read more
The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are ... Read more
A cholesterol-lowering medication, bezafibrate, taken daily for 12 weeks did not induce the formation of new mitochondria in people with mitochondrial myopathy ... Read more
GenSight Biologics announced it has finished a pre-submission meeting with the European Medicines Agency (EMA), a key step in filing a request for ... Read more