Wednesday, April 30, 2014

Longitudinal Study of the Porphyrias

Longitudinal Study of the Porphyrias
We continue to share ongoing porphyria studies, please consider being a part of them.
The 7201 Longitudinal Study of the Porphyrias is a longitudinal multidisciplinary investigation of the human porphyrias including the natural history, morbidity, pregnancy outcomes in people with these disorders. We expect to enroll at least 600 incident cases during the 5-year study period.
The purpose of this long-term follow-up study is to provide a better understanding of the natural history of porphyrias, as affected by available therapies, and to aid in developing new forms of treatment.
The general research questions are:
What is the prevalence of specific indicators of disease severity, including neuro visceral and cutaneous manifestations, levels of porphyrin precursors and porphyrins, quality of life, long term effects on the liver, kidneys and other tissues, and case-fatality associated with the various forms of porphyria?
What are the relationships between disease severity and progression and various biological characteristics, contributing endogenous and environmental factors and levels of porphyrin precursors and porphyrins?
What is the safety and efficacy of currently used and new therapies for the human porphyrias, especially in the long term?
In order to participate in a study, please contact us at the APF 866-apf-3635. For research studies sign up please email Amy Chapman at

"Remember....Research is the key to your cure!"

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