Research study is open to participants worldwide to advance understanding and treatments for rare disease causing blistering that may be accompanied by severe pain, itching, burning, and stinging.
Sacramento, CA and Washington, D.C., March 31, 2017—The International Pemphigus &… Read More
Washington, D.C., March 15, 2017—The National Organization for Rare Disorders (NORD) today announced the people, organizations, and innovators who will be honored at the 2017 Rare Impact Awards on May 18 in Washington, D.C.
The Rare Impact Awards is a… Read More
Danbury, Conn., February 28, 2017—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, announced a year-long awareness campaign for rare disease.
Kicking off today—Rare Disease Day 2017—“Do… Read More
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
Research study is open to participants worldwide to advance understanding and treatments for rare disease causing non-syndromic intellectual disability ranging from mild to severe with attention deficits, impulsivity, and/or mood disorders. Seventy to eighty percent of children with SYNGAP1 also have some type of epilepsy and the SYNGAP1 gene has… Read More