Even though Gaucher disease is one of the better known rare diseases and diagnostic testing is available, some patients still go for 10 years or longer without getting an accurate diagnosis after symptoms first appear. read more >
One of my favorite moments at NORD’s recent 30th Anniversary celebration was seeing Abbey Meyers and Congressman Henry Waxman reconnect. The two played leading roles in the drama that resulted in the Orphan Drug Act of 1983. Both went on to provide key leadership on related issues — Abbey as the president of NORD for its first 25 years and Rep. Waxman in Congress.
Today is the day when people around the world will be posting stories, photos and videos about what it means to live with a rare disease. From Kalamazoo to Kiev, the last day of February is observed as Rare Disease Day. read more >
The fourth annual Raise Your Hand campaign launched this week, and it’s already creating a social media buzz. This campaign, sponsored by Lundbeck, raises awareness and support for rare disease research. It also gives a spark of hope to patients previously left on the sidelines in the quest for a treatment. read more >