Washington, D.C., January 11, 2017—Data released by the U.S. Food and Drug Administration (FDA) show that 9 of the 22 new medications approved in 2016, or 41 percent, are orphan drugs to treat rare diseases.
“The percentage of orphan drugs approved in recent years by FDA remains high and this is welcome news for the 30… Read More
The Hill, a top U.S. political website, read by Congressional offices, the White House, political pundits, and more, today published the following op-ed by NORD President and CEO, Peter L. Saltonstall, urging Congress to reauthorize the Rare Pediatric Disease Priority… Read More
Debra Miller co-founded CureDuchenne in 2003 with her husband, Paul, after their only son was diagnosed with Duchenne Muscular Dystrophy. With a background in sales and marketing, she leads CureDuchenne and has funded seven research projects that have advanced to human clinical trials, three of which are the… Read More
Next week, a Food and Drug Administration panel of experts will hold the first of two meetings to review two drugs designed to slow the muscle wasting effects associated with Duchenne Muscular Dystrophy (DMD). These much-anticipated events provide hope for… Read More
When Robert and Theresa Capolongo were preparing to be parents, many words were thrown around by their friends and family about the joys of having children. “Fulfilling.” “Life-changing.” “Beautiful.” These were, of course, balanced with considerate pieces of advice. “It’s the toughest job out there.” “It’s not about you anymore,… Read More