Over 20 million people in the U.S. live with a rare disease, and many face years of uncertainty and delayed diagnosis. The National Academies’ Forum on Advancing Diagnostic Excellence will host a public webinar on November 6 exploring opportunities to improve rare disease diagnosis and the patient journey towards a timely and accurate diagnosis. Discussions … Read more
The National MPS Society will host a timed 5K run and 1 mile walk, beginning at 9 AM on Saturday, March 28. The 1 mile walk will begin at 9:05 AM. Packet pickup and day of registration will open at 7:30 AM. A picnic for attendees and volunteers will be held after the race! This … Read more
Advancing treatments for rare diseases requires innovative ways to measure success. Join FDA, researchers, clinicians, and innovators for a free, one-day virtual workshop open to the public focused on emerging scientific and regulatory pathways for advancing surrogate endpoints in rare disease drug development. This workshop is being convened in support of the Rare Disease Endpoint … Read more