NORD publishes 20 rare disease patient groups in FDA cooperation
NORD is the top independent nonprofit organization that aims to help families and people who are afflicted with rare diseases. Now, the 20 patient groups will be included in developing a natural history study with NORD’s research platform.
“Our goal is for the one in 10 Americans with rare diseases, most of whom are children, to have a treatment and cure, and we developed NORD’s Natural History Study platform to eliminate challenges standing in the way of that target,” Peter Saltonstall, NORD president and CEO, said. “We thank the FDA for its support of NORD and ongoing commitment to rare diseases. NORD’s Natural History Studies project empowers patients and families to help eliminate some of the ‘I don’t know’ in rare disease research, making way for progress.”
The 20 groups include Organic Acidemia Association, Hereditary Neuropathy Foundation, XLH Network Inc., Pitt Hopkins Research Foundation, CCHS Family Network, Platelet Disorder Support Association, The OMSLife Foundation, APS Type 1 Foundation, Global Foundation for Peroxisomal Disorders, Desmoid Tumor Research Foundation, Galactosemia Foundation, International Pemphigus & Pemphigoid, Scleroderma Research Foundation, Adult Polyglucosan Body Disease (APBD) Research, The Morgan Leary Vaughan Fund, United Leukodystrophy Foundation, Bridge the Gap-SYNGAP Education and Research Foundation, American Multiple Endocrine Neoplasia Support (AMENSupport), Worldwide Syringomyelia & Chiari Task Force and Lipoprotein(a) Foundation.