Skip to main content

Porphyria Post

Harvoni Study - PCT
Do you have PCT? Are you interested in participating in research? Do you have Hepatitis C? If you answered YES to these questions, this is for you.
We need YOU for a clinical trial!
The purpose of this clinical trial is to assess whether Harvoni alone is an effective therapy in active PCT patients with Chronic Hepatitis C.
Who can participate?
·       Adult patients with PCT who also have Hepatitis C
If you are interested in participating please contact Edrin Williams, Director of Patient Services at the APF office at 301.347.7166 for additional information.

PRIORITY REVIEW letters sent to the FDA!!

You are an amazing community! We had a great response to the call urgent call to action for patient letters.  511 letters, a petition, and many poignant photos were sent to the desks of decision-makers at the FDA requesting Priority Review for SCENESSE (afamelanotide 16mg). 
The new drug application for this treatment was submitted to the FDA on June 22.  The FDA has sixty days to decide if the filing will receive Priority Review (6 months) or Standard Review (10 months).  Our EPP patients have waiting long enough for treatment.  We hope receipt of these letters will help urge the FDA to select the quicker path to treatment.  Nearly 6,700 doses have been implanted in over 800 patients. 
WANT TO DO MORE? Check out these In-District Lobby Days below and call your legislators to ask them for help. 

You can participate in In-District Lobby Days!
From July 31st through September 4th, rare disease advocates from across the country will surge into the district offices of their Members of Congress to advocate for legislation benefiting the rare disease community.
The registration deadline for this event has passed. However, you can still contact your legislators offices while they are in-district.

The American Porphyria Foundation can help you with issues and talking points important to each type of porphyria.

EPP Clinical Trials – Participants Needed!
Please contact the APF on 866-APF-3635 to request information on these new clinical trials.  There are seven study sites across the US that need your participation. Do you want a new treatment for EPP? – then come be part of the solution. 
Remember…Research is the Key to Your Cure!! Every step along the way is important.


Popular posts from this blog

Amazing patient advocacy....and needed media for this issue. Thank you Terri Witter!


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…