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.
I represented NORD at the White House this morning at an event where President Obama announced the launch of a “Brain Initiative” to help researchers find new ways to treat and cure brain disorders. The initiative includes: read more >
Now that the election is well behind us, talks have resumed in Washington, DC about the Federal budget. To recap — the only major piece of the larger budget puzzle that was addressed at the end of the year was the expiration of the current Federal income tax schedule, also known as the ‘Bush tax cuts’ as they were championed and signed into law by former President George W. Bush. read more >
Posted at January 15, 2013 5:11 pm by Marsha Lanes
Whole genome sequencing (WGS) is the genetic test that determines the order of all 3 billion letters in a person’s DNA, and is a technology that has become well known to the rare disease community. It can reveal not only the genes responsible for production of an abnormal protein associated with a disease, but also the presence of other abnormal genes or variations in gene structure and regulation. Success stories describing the use of this technology to help some individuals with rare diseases overcome the “odyssey of diagnosis” have been widely reported (links below) with understandably great enthusiasm, and this technology holds enormous promise for continuing to uncover the underlying causes of rare diseases. read more >