This blog is dedicated to all the Porphyria patients worldwide.
The American Porphyria Foundation will provide updates and information here, as well as on the main site - http://porphyriafoundation.org
In preparation for Porphyria Awareness
Week (April 6-13), the American Porphyria Foundation invites you to share
your story with the porphyria community. With permission, all stories will
be featured on the APF Website. We will also feature select stories in our
Stories should be
about 500 – 2000 words and full of your personality. Make sure to include
information about your specific porphyria type, your diagnostic journey and
how this disease has impacted your life.
We will be sending
out a small token of our appreciation to the first 5 submissions.
We look forward to
receiving your stories. The APF office hears regular feedback that the
stories listed on our site have helped them feel less isolated and work
toward diagnosis. Thank you for having a hand in helping others through
your stories and words.
The APF will be
exhibiting at the 2019 ACMG Annual Meeting in Seattle, WA April 3-5.
Are you interested in volunteering at our booth? If so, please contact
Edrin Williams, Director of Patient Services, for additional information at
email@example.com or 301.347.7166.
This is an
incredible opportunity to raise awareness about Porphyria to those with
little knowledge about this rare disease.
Pharmaceuticals announced yesterday that Scenesse has been granted
Priority Review by the FDA - the New Drug Application will be reviewed by
July 8, 2019. Visit www.clinuvel.com for
The APF and you,
the patient community, have been relentless in pursuit of this
treatment. Writing letters, making calls, signing petitions, sending
photographs, attending meetings - it worked!
We will continue
to focus on this until this treatment is approved and available to all
EPP patients in the US.
for FIND YOUR SHADOW 2019! The application will be on the APF
website starting this Friday, January 11th!
Once again this year, the Shadow Jumpers
program will be selecting one family for an all-expenses paid trip to
Walt Disney World in Orlando, FL. All children ages 5-17 and their
families are eligible to apply. Shadow Jumpers is dedicated to finding
creative ways to help kids and their families to take on a safe
In 2018, the Stuhlsatz family was
selected for Light the Moment 2018, the theme of last year's
trip. Their trip - from beginning to end - was "a once in a
is excited for your submissions to FIND YOUR SHADOW 2019!
*please note: Shadow Jumpers is funded
through donations and charitable contributions separate from the APF.
Panhematin Study -
Do you have AIP, VP or HCP? Are you interested in
participating in research? If so, we are recruiting volunteers for the
Our researchers need people to volunteer for the Panhematin
study. Completing this study will help prevent insurance companies from not
paying for this treatment.
The purpose of this study is to determine if Panhematin is
safe and effective for prevention of acute attacks of porphyria.
you predict when your next attack will happen?
you currently receiving prophylactic heme treatment?
Please contact Edrin at the APF office (301.347.7166) if you
are interested in participating.
Trials: Volunteers Needed!
We are close, but we need YOU!
A clinical trial with an oral investigational drug intended
to reduce phototoxicity in people living with EPP is underway.
Please note that a Phase 3 study is very unlikely if Phase
2 is not completed. To date, this drug has proven nontoxic and there
have been essentially no dropouts in the Phase 2 study to date.
This is an oral
drug which makes administration simple.
This study in
adults will pave the way for a near-future pediatric trial. EPP is a rare
disease and patients must participate in order to get the drug
To date there is no public information on the availability
of Scenesse, and if approved, two drugs on the market may lower their cost.
We will connect you with a Research Coordinator that will
answer all your questions and concerns. We will work to make this
process as easy as possible for you.
**Note: If you have not been contacted by a research
coordinator please reach out to the APF office at 301.347.7166. **
diagnosis of EPP
written informed consent to participate
willing and able to travel to all study sites for scheduled visits
- 70 years of age at the time of screening.
Sinai - New York City
Forest Baptist University - Winston Salem, NC
of Miami - Miami, FL
of Texas Medical Branch - Galveston, TX
of Utah - Salt Lake City, UT
of California San Francisco - San Francisco, CA
We need YOU!
Please dig deep and consider being part of this changing moment for
all with EPP.
Contact us here at the APF office to get
in contact with a research coordinator.