The National Organization For Rare Diseases (NORD) recently announced the twenty rare disease patient groups selected to develop natural history studies, supported in part by a cooperative agreement with the U.S. Food and Drug Administration (FDA).
The Hereditary Neuropathy Foundation is proud to be included among leading rare disease groups, as we collectively aim to fill the research gaps to better understand how these rare diseases progress over time.
With nearly 7,000 rare diseases and disorders, 95 percent of which have no treatment, a collective initiative is needed to help researchers and drug developers eliminate the obstacles they face in finding treatments. Small patient populations, lack of data, unclear clinical endpoints, and clinical trial enrollment and retention make finding treatments for rare diseases especially challenging.
NORD created the Natural History Study platform to help meet these challenges head-on.
Natural history studies are studies that follow a group of people over time who either have, or at risk of having, a specific medical condition or disease. Natural history studies of rare diseases can produce the vital information needed for clinical trial design. Information such as biomarkers, demographics, clinical symptoms, genetic and environmental variables, and patient perspectives are essential to helping researchers better understand disease progression.
NORD President and CEO Peter L. Saltonstall states:
“Our goal is for the 1 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.”
“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.”
According to NORD, the groups were selected by a competitive application process reviewed by an internal committee. Representative diseases must have diagnostic challenges, limited or no research, and cover a broad range of symptoms and medical specialties, including neurology, cardiovascular, musculoskeletal, immunology, and endocrinology. Selected organizations are all members of NORD.
HNF is excited to be a part of NORD’s Natural History Studies Project. Follow us on the HNF Facebook Page or sign up for our CMT Update newsletter to stay current on the latest developments of this initiative.
I have cmt type 6 along with my mother and 3 siblings. More information on this disease would be great as we have never really been given any!
Hi Jarman, HNF has a research strategy for CMT6. Do you know your mutation? I would suggest you contact us via email at email@example.com. Thanks for posting and sharing.