Shapiro & Delaney presented the poster "Importance of Common Data Elements for Rare Disease Clinical Trials."
WORLDSymposium 2016
12th annual research meeting
February 29: “Emerging Trends” (State of the Art for Experts)
March 1–3: Research Meeting
Visit WORLDSymposia's Website
FDA Workshop
Held April 16 and 17, this FDA workshop focused on cognitive endpoints in rare inborn errors of metabolism and common diseases. These presentations (video, transcripts, and slides) are now on-line.
UK Mucopolysaccharidosis Society Workshop
The UK Mucopolysaccharidosis Society had an important recent workshop regarding natural history studies and clinical trials in Sanfilippo syndrome (Mucopolysaccharidosis Type III) that included academia, industry, and advocacy groups. This workshop concluded that common natural history databases and standard protocols were necessary to ensure that patients obtain the best services.
NORD: National Organization for Rare Disorders
The next Rare Disease Day is February 29, 2016. Information available at NORD regarding other rare disease patient advocacy groups.
Visit the NORD Website
NORD Membership List
