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Join Research and the Porphyria Registry

Join Research and the Porphyria Registry


There are two main types of clinical studies: clinical trials and observational studies.  In a clinical trial, there is some form of treatment intervention. There is no intervention in an observational study, which is aimed at observing patients to better understand the long-term course of their disease.
Clinical trials are used to test new treatments before they are approved for use by the FDA. This type of trial gives patients a chance to try out a new medication in its early stages. As with any experiment, the result of a trial is not known before its conclusion. Your participation could help demonstrate a terrific treatment breakthrough, or it could help scientists discover that a new treatment does not work after all. There may be some risk involved from the treatment in a clinical trial.
Participating in either a clinical trial or an observational study is a serious responsibility. Volunteering to participate could be a way to help yourself, affected family members and other patients by advancing medical and scientific knowledge of your condition. Some patients derive great satisfaction from assisting doctors in the study of their disease. Participation in a study can also mean a chance to meet a porphyria researcher in a clinical setting, and the consultation can be beneficial.
For information on porphyria trials currently recruiting patients, call the APF office or visit NIH’s clinical trials website: “Clinical Trials of Medical Treatments: Why Volunteer?” might also be useful reading as you think about whether you’d like to participate or not.

Join the Porphyria Registry and LET THE GOVERNMENT KNOW THEY MUST PROVIDE FUNDING FOR PORPHYRIA RESEARCH!!!!!
To join the Contact Registry, click here to open a page that lists all of the rare disease consortia. Scroll down the page until you come to the Porphyria Consortium and click on your type of porphyria. You will then be asked to complete a simple form including information about the date of your diagnosis, if you know it. If you have copies of your initial diagnostic lab results, you may want to have them handy when you go to the registry website.
Porphyria experts have created this National Porphyria Registry—a type of partnership between doctors and patients— as a way for those with porphyria to share information about their health and treatment so physicians can learn from their experience and use that knowledge to enhance diagnosis, treatment and eventually find a cure for porphyria.
It is the best means to prove that there are enough porphyria patients who want improved health care. If we don't speak up, we will be left behind when research funding is given. We DO NOT HAVE ENOUGH PEOPLE ON THE REGISTRY. Please join the registry.
Joining the Porphyria Registry is anonymous and free of charge. All data will be stored in a secure, computerized database. No personal identifying information (such as your name, address, telephone number) will be given to anyone without your expressed approval.
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The registry is not linked to APF membership, but we hope you will join the American Porphyria Foundation too! So please consider joining the Contact Registry, and thank you for continuing to be a member of the APF.
Doctors who study rare diseases see a relatively small number of sufferers over many years of practice. This Registry will give a big boost to medical and scientific understanding of porphyria by bringing together information from patients all over the country.
If you need help enrolling in the registry contact our office toll free at 1-866-APF-3635.

https://www.rarediseasesnetwork.org/registry

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Q & A WITH PORPHYRIA EXPERT, DR. BRUCE WANG, UCSF

Q & A WITH PORPHYRIA EXPERT, DR. BRUCE WANG, UCSF 
The APF asked our Facebook friends for their top questions they would ask a porphyria expert.
 The following questions were submitted to Dr. Wang for his responses ... Q. Does EPP give us bad teeth? Also, do people with EPP get stomach pains or is that with the other porphyias? A. The porphyrin that accumulates in EPP patients is protoporphyrin IX, which does not cause discoloration to teeth or abdominal pain.
 The type of porphyria that leads to discolored teeth is Congenital Erythropoietic Protoporphyria. The porphyrias that lead to episodic abdominal pain attacks are the acute hepatic porphyrias. Q. I have EPP and I have a severe reaction on my hands and lips. Do I seek urgent care? Also, what can you even do when you burn your lips? A. The acute reactions to sunlight in EPP can be very severe and, unfortunately, there are not many effective options to treat the symptoms. Nonsteroidal anti-inflammatory drugs (NSAIDS such as ibup…