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Showing posts from August, 2019

Patient Education Programs

Patient Education Programs

The American Porphyria Foundation is dedicated to the health and well-being of individuals affected by Porphyria. One way that the APF does this is by Patient Education Programs

Maintain, update, and expand comprehensive website, brochures, pamphlets, books and educational materials for each type of porphyria, genetics and research.Develop new educational programs and services for purposes of research, treatment, diagnosis.Distribute materials on porphyria treatmentEngage and assist in Health Insurance assistance and billing problemsUpdate relevant new information Update on RESEARCH- ALL THE TIMEParticipate in convention exhibitsDevelop & deliver patient education programs for physician relationshipsEducate the FDA drug approval processEducate on congressionional processPromote research process and participationArrange conference calls with expertsProvide expert consultation with patients and physicians Members, 
We cant do this alone.  If you need any ty…


Ongoing interactions between the US Food and Drug Administration (FDA) and CLINUVEL’s scientific teams continue to take place as the final outcome date of October 6, 2019 approaches. This date has alreayd been extended from May 31st to October 6th. In the latter half of the scientific review, the FDA will attempt to address substantive review issues which have not yet been covered by its committee members. At this stage, the FDA will use its time to gain a broader insight of risks and benefits while reserving the right to identify new review issues that may trigger an extension of the Prescription Drug User Fee Act (PDUFA) goal date. There is no indication that the current PDUFA date cannot be met; it is CLINUVEL’s expectation that October 6th will remain the current PDUFA goal date for an outcome to be provided.
Read Clinuvel's August Newsletter at…/20…/08/CLINUVEL-Communique-VI.…

101 Acute Hepatic Porphyrias 3 of 3

Treatment During Attacks Pain can be the primary and only complaint of a patient in a porphyric crisis and sometimes for some time beyond. Appropriate pain measures should be applied and it would be unwise and not good medicine to overrule a patient by withholding medication. Hospitalization is often necessary for acute attacks. Medicine for pain, nausea and vomiting along with close observation are required. Initial treatment of AIP attacks consists of stopping harmful drugs the patient may be taking and providing a high intake of carbohydrate (300 grams or more per day). Carbohydrate can be given either in the form of an oral carbohydrate or by intravenous infusion. Intravenous infusion is better in moderate or severe attacks or for patients who are unable to ingest enough carbohydrate orally. Pain, anxiety and emotional symptoms should be treated with safe drugs. Attacks with muscle weakness occasionally require respiratory support, but this is unusual unless an attack is brought …

101 Acute Hepatic Porphyria 2 of 3

IF THERE IS A CLINICAL SUSPICION OF PORPHYRIA Click to enlarge chart Please note: the rapid PBG test kit is no longer available Clinical Features of AIP Signs and symptoms of AIP usually occur intermittently and include abdominal pain, constipation, muscle weakness, pains in the arms and legs, insomnia, emotional difficulties, rapid pulse, and high blood pressure. Muscle weakness can be severe during a prolonged attack due to the effects of Porphyria on nerves that control muscle. In fact, all symptoms of AIP appear to be due to effects on peripheral nerves, the nerves in the abdomen or the central nervous system. Precisely how Porphyria produces pain and other symptoms related to the nervous system is not yet well understood. PBG is produced in excessive amounts by the liver in AIP, but it has not been proven that excess PBG can damage nerve tissue. Because Porphyria is rare and its symptoms often suggest other diseases, the correct diagnosis is often delayed. Attacks due to harmful …

Physician Education Packets

Physician Education Packets –
Did you know that the APF will send a complimentary physician education packet to any of your treating doctors? Primary Care, Internist, Family Medicine, Hematologist, Oncologist, Gastroenterologist, etc..
If you want all your physicians invested in your care, let us help you educate them about porphyria. These complimentary packets are tailored to be specific to your type of Porphyria.  As an example, the acute porphyria packets include information about the intensity of acute porphyria pain.  The EPP packet includes information on the visible spectrum of light triggering phototoxicity.  Email Edrin Williams, Director of Patient Services to request your packet(s) to be sent today! Please include their name and physical location.

Thanks,  Edrin
Edrin R. Williams, MHSA
Director of Patient Services
American Porphyria Foundation
4915 St. Elmo Ave.,Suite 200
Bethesda, MD 20814
301.347.7166 office email

101 Acute Hepatic Porphyrias 1 of 3

AIP, HCP, VP & ADPPlease note that the symptoms and treatments for AIP are applicable to Variegate Porphyria (VP), Hereditary Coproporphyria (HCP), and ALAD Porphyria (ADP). Unlike AIP and ADP patients, however, VP and HCP patients often develop photosensitivity. Acute Intermittent Porphyria (AIP) is a rare disease that is usually inherited from one parent. It is almost always latent (i.e. does not produce illness) in childhood and is usually latent in adults throughout life. When the disorder is active, it can cause intermittent attacks of abdominal pain as well as a variety of symptoms, which range from mild to life-threatening. After the correct diagnosis of AIP is made, simple precautions can be taken to prevent attacks. It is important to check all relatives of Porphyria patients for the genetic defect, so they can take the same precautions and avoid becoming ill from Porphyria. Even relatives who have never had symptoms should be tested, as described below. Diagnosis Labora…

NIH-Tips for Finding Financial Aid

Tips for Finding Financial AidEn EspaƱolListen The cost of care for rare medical conditions can place a significant financial burden on families. The following resources may help you find the financial support you need.

Does the National Institutes of Health (NIH) provide financial assistance?The NIH is the nation’s medical research agency. The NIH is not authorized to provide direct financial assistance but can provide medical care to participants who are enrolled in a clinical research study.

See our guides on How to Get Involved in Research and Help with Travel Costs for more information about participating in clinical trials and finding financial assistance with the cost of travel.

In the following sections, you will find nonprofit, government, and community resources that can help with financial assistance. We also encourage you to search the GARD website using the name of your condition. On the GARD disease pages you can find resources in the “Organizations” and “Living With” sect…