NORD-FDA Natural History Study Frequently Asked Questions What is the NORD Registry Program? The NORD Registry Program is a compilation of services that assist member organizations through all the steps of developing, launching and running a Registry. What is the NORD Natural History Study Database Project? The NORD Natural History Study Database Project is one […]
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Jan. 15, 2015 TOPIC: Research Registries for Rare Diseases: Involve the Patient Registries for Rare Diseases: Involve the Patient Posted by Jennifer Huron In a new interview with Medscape, Marshall L. Summar, chief of genetics and metabolism at Children’s National Medical Center in Washington, D.C. and NORD board member, talks about the importance of patient registries for rare diseases,
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https://rarediseases.org/wp-content/uploads/2015/05/BioCentury-pg5-6.pdf CHARTING RARE STARS BY EMILY CUKIER-MEISNER, SENIOR WRITER A databank constructed by the National Organization for Rare Disorders and the VHL Alliance could serve as a model for how patient organizations can generate natural history data to help speed development and FDA review of new treatments. The Cancer in Our Genes International Patient
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https://blogs.fda.gov/fdavoice/index.php/2014/10/the-more-we-know-about-rare-diseases-the-more-likely-we-are-to-find-safe-and-effective-treatments/ The more we know about rare diseases, the more likely we are to find safe and effective treatments Posted on October 23, 2014 by FDA Voice By: Janet Woodcock, M.D. You may be inclined to think that rare diseases affect only a tiny fraction of the more than 320 million people in our country. That’s true about
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NORD-FDA Natural History Study Project Page NORD is partnering with disease specific advocacy organizations to launch patient-centered registries to gain a better understanding of rare diseases. The registries will allow patients around the world to share relevant data, connect with researchers, and support research on how variables such as diet, exercise, environmental factors and other
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From the Editor… As a rule, PRP patients and caregivers are given the proper spelling of pityriasis rubra pillars, the acronym “PRP” and a referral to Dr. Internet. Some of us reach out to Dr. Yahoo. Others seek out Dr. Google. It doesn’t take long for us to learn that using PRP doesn’t work. Enter “PRP” and the
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From the Editor… Cami B (Pine City, MN) is participating in ongoing PRP research at Thomas Jefferson University. Misdiagnosed She received an email on June 13, 2017 requesting blood samples. from TJU researchers. The following day she went to the lab where the blood samples were taken. It was then off to the Post Office.
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NOTES Describe Juvenile Onset PRP ✽ We want to describe Juvenile Onset PRP rather than PRP in general ✽ This should be a general overview and include Type 3, 4 and 5 ✽ What are the SPECIFIC challenges facing PRP parents and PRP children? Describe your child’s version of PRP ✽ We want to describe the child’s SPECIFIC
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The many definitions of REMISSION There does not appear to be an official definition of REMISSION as it applies to pityriasis rubra pilaris. REMISSION comes from the Latin remissionem, meaning “relaxation, a sending back.” If something difficult lets up, you’ll be able to relax a little. Here are some definitions to ponder. When you are
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Dateline: November 22, 2022 — This page of the PRP Survival Guide was originally designed to be a repository of experiences and insights shared by the parents/caregivers of children diagnosed with PRP. It was also intended to serve adults diagnosed with PRP as children. With the help of PRP parents, we hope that this webpage will
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