Skip to main content

Posts

Access to Care Toolkit for the Acute Porphyrias is now available Part 1

What's all in the New Acute Porphyria Tool Kit?  (AHP)

How can it benefit me or a caregiver?  How will it affect my Doctor's appointments?

All these tools come free of charge and can be downloaded any time.  

Please click on the link here: Access to Care Toolkit:  
http://www.porphyriafoundation.com/content/aip-hcp-vp-access-care-toolkitAccess to Care Toolkit for the Acute Porphyrias is now availableA downloadable Access to Care Toolkit is a resource designed to help patients living with Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP), and Variegate Porphyria (VP) or their caregivers, loved ones and healthcare providers secure access to Panhematin at their preferred health facility.  We have recently learned of patients who are being denied this treatment from some hospitals and directed to secure another healthcare provider.  If this has happened to you or someone you know or care for, please use these tools to request help from your state and local representa…
Recent posts

National Porphyria Awareness Week (NPAW) is right around the corner.

National Porphyria Awareness Week (NPAW) is right around the corner.NPAW: April 22 - 29, 2017See the story below as a great example of how to raise awareness in your community. For the second year in a row, the Cook family put on a fantastic benefit barrel race.  As you may know, the Cook brothers, Cason and Caul, have EPP and have set a great example about enhancing awareness of the disease  in their local area.  They have been hosting a Hat Day,

Access to Care Toolkit for the Acute Porphyrias is now available

Access to Care Toolkit for the Acute Porphyrias is now availableA downloadable Access to Care Toolkit is a resource designed to help patients living with Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP), and Variegate Porphyria (VP) or their caregivers, loved ones and healthcare providers secure access to Panhematin at their preferred health facility.  We have recently learned of patients who are being denied this treatment from some hospitals and directed to secure another healthcare provider.  If this has happened to you or someone you know or care for, please use these tools to request help from your state and local representatives and health advocacy organizations.  We understand the debilitating effects of acute porphyria and hope these resources will help you secure access to Panhematin when you need it most.
The Toolkit contains the following materials:*Healthcare Conversation Tracker is a simple form to record your conversations with doctors, insurance agents…

Alnylam Receives European Medicines Agency PRIME Designation for Accelerated Assessment of Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyrias

Alnylam Receives European Medicines Agency PRIME Designation for Accelerated Assessment of Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyrias 03.01.2017CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, announced today that the European Medicines Agency (EMA) has granted access to its Priority Medicines (PRIME) scheme for givosiran (ALN-AS1), an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias. The purpose of the PRIME initiative is to bring treatments to patients faster by enhancing the EMA's support for the development of medicines for diseases where there is an unmet medical need and where early clinical data show potential to benefit patients. Promising results from the Phase 1 study of givosiran formed the basis of the application for PRIME. The ongoing Phase 1 trial is being conducted as…

Why Join a Clinical Study?

Clinical StudiesThere 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…

The PCT Story of Kelly Story

Kelly StoryType of Porphyria: Porphyria Cutanea Tarda (PCT)
My name is Kelly Story and I live with my husband, Chad, and two cats in Kissimmee, FL. In July of 1999, one month after our wedding, I was out of town on business, and I noticed tiny little water blisters all over my hands. The blisters didn't itch and were not painful. Although I found them to be very odd, I didn't worry much.Over a short period of time, my skin became extremely fragile. It seemed like almost anything would cause a scrape on the skin of my hands. Plus, the blisters got much worse. My hands looked like something out of a horror movie. I was so embarrassed all of the time, and I cried a lot. I went to a dermatologist. After two visits, he told me that he was fairly certain that I had PCT but suggested that I go to a specialist. After several humbling tests and weeks of waiting, I was officially diagnosed with PCT.The doctor said that I would have to start phlebotomies. This meant that over a pint of bl…

Porphyria cutanea tarda (PCT

Porphyria cutanea tarda From Wikipedia, the free encyclopedia For the song by AFI, see Black Sails in the Sunset.Porphyria cutanea tardaClassification and external resourcesSpecialtyendocrinologyICD-10E80.1ICD-9-CM277.1OMIM176100176100DiseasesDB1037629123