Friday, June 29, 2018

Family, Friends, Spouse of an EPP Hero Please Write FDA a Letter

THANK YOU TO ALL FOR YOUR LETTERS REQUESTING PRIORITY REVIEW FOR SCENESSE!
They are starting to pour into the APF office. Haven't taken a moment to write yet...now is the time. Don't delay.
ADDRESS TO:
Scott Gottlieb, M.D.
FDA Commissioner
U.S. Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD 20993
Dear FDA Commissioner:
MAIL TO:
American Porphyria Foundation
4915 St. Elmo Avenue, Suite 105
Bethesda, Maryland 20814
OR EMAIL TO:
porphyrus@porphyriafoundation.com

Porphyria POST regarding Letters & Trial Info

You can be a medical hero! A new investigational drug for EPP is ready for clinical trial. All we need to begin is YOU! The trial spots will fill up fast…join now! Contact the APF on 1-866-APF-2635 or email
porphyrus@porphyriafoundation.org to be connected to a study site. Don’t wait! Remember…Research is the
Key to Your Cure!


An oral investigational drug has been developed with the potential to increase sunlight duration and tolerance in
individuals with EPP. This phase II clinical trial is needed to understand if the investigational drug works and to
ensure that it is safe. The study duration is 24 weeks including follow-up. You must be age 18-70 to participate
and have a confirmed diagnosis of EPP. All travel is included and will be arranged by a concierge service.


"Remember...Research is the key to YOUR cure!"


Each Step Toward Finding an Effective Treatment is Important!


Porphyria Post
Request
The New Drug Application for Scenesse has been submitted to the FDA.
Now is the time to work hard as patient advocates.
The APF is launching a new campaign to request Priority Review
Now is the time to send your letter requesting priority review for Scenesse. We need to move Scenesse, a long-awaited treatment for EPP, through the regulatory process as quickly as possible. Now that a complete new drug application has been submitted to the FDA - they have the option of granting priority review which moves the process faster. Please share with the FDA...
* That there is no treatment for EPP
* That there is an unmet medical need for a treatment for EPP
* That living with EPP is a tremendous burden
* How living with EPP affects your quality of life
It is important that you send your signed letter to the APF immediately.
Begin your letter:
Scott Gottlieb, M.D.
FDA Commissioner
U.S. Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD 20993
Dear FDA Commissioner:
Mail your letter to:
American Porphyria Foundation
4915 St. Elmo Avenue, Suite 105
Bethesda, Maryland 20814
Alternatively you can scan your signed letter and email it to porphyrus@porphyriafoundation.com.
All letters must be signed.

We will present all of the letters at once to the FDA!
Please send letters today or ASAP! Time is of the Essence!

Study # 7210 Newer Direct-Acting Anti-Viral Agents as Sole Therapy of Porphyria Cutanea Tarda in Subjects with Chronic Hepatitis C

Newer Direct-Acting Anti-Viral Agents as Sole Therapy of Porphyria Cutanea Tarda in Subjects with Chronic Hepatitis C

In order to participate in a study, you must personally contact 
the APF Edrin 1/866/APF/3635
For Diseases:
  • Porphyria Cutanea Tarda (PCT)

Background

Many people with PCT also have hepatitis C virus (HCV). There have been reports that the treatment of HCV infection can also resolve the symptoms of PCT. However, this has not been clearly established.
Therefore, the purpose of this study is to determine if giving a standard treatment for HCV is also an effective treatment for PCT.
The research aims are:
  1. To assess whether Harvoni (the treatment for HCV) alone is an effective and durable treatment for PCT in patients with both HCV and PCT.
  2. To determine whether treating patients with PCT and HCV with Harvoni is as effective as treating PCT with the standard therapies, phlebotomy or hydroxychloroquine.
This is a clinical trial, which means its purpose is to study an intervention or treatment. In this study all patients with PCT will be given a standard dose of Harvoni and monitored for two years. Currently there are two standard therapies for PCT, phlebotomies (removing certain amounts of blood at specific intervals), or low dose hydroxychloroquine (an oral pill). These treatments are used for patients with PCT whether or not they also have HCV. For patients with HCV however, we do not know whether treating the HCV first will also resolve the PCT symptoms.
There will be an initial visit to determine whether participants are eligible to be in the study. If a participant is found to be eligible, he/she will be asked come to the study site once every month over the course of one year, and then once every 3 months for an additional year. There will be approximately 17 visits over the course of the whole study. At these visits the study doctors will check in with the participant and some blood and urine samples will be taken. Participants will not be charged for any of the lab tests that are being done as a part of this study alone. All participants in this study will receive the Harvoni pills at no cost to them.

Targeted Enrollment

To be eligible to participate, you must:
  • Have a biochemical diagnosis of Porphyria Cutanea Tarda (PCT)
  • Have the typical symptoms of PCT (blistering in response to sun exposure, fragile skin, etc.)
  • Be at least 18 years old
  • Have chronic hepatitis C virus (HCV)
  • If you are female of child-bearing potential, you must be willing to take an effective method of contraception
You are not eligible to participate if you:
  • Are pregnant, planning to become pregnant, or breast-feeding
  • Used any other clinical or experimental therapy for PCT in the past month
  • Are currently enrolled in the Porphyrias Consortium study “Therapeutic Studies of PCT”
  • Have an HIV infection that is not well controlled
  • Drink more than 25 alcoholic drinks in a week
  • Have been taking a medication called amiodarone within 60 days of enrolling in this study
  • Have been taking St. John’s wort within 28 days or enrolling in this study
  • Have uncontrolled diabetes
  • Have chronic hepatitis B
  • Have other liver disease or have liver cancer
  • Have had a liver transplant in the past
  • Another disease or condition that the study doctors judge would interfere with the study
In order to participate in a study, you must personally contact the

Thursday, June 28, 2018

PORPHYRIA POST URGENT

Porphyria Post
Request
The New Drug Application for Scenesse has been submitted to the FDA.
Now is the time to work hard as patient advocates.
The APF is launching a new campaign to request Priority Review
Now is the time to send your letter requesting priority review for Scenesse. We need to move Scenesse, a long-awaited treatment for EPP, through the regulatory process as quickly as possible. Now that a complete new drug application has been submitted to the FDA - they have the option of granting priority review which moves the process faster. Please share with the FDA...
* That there is no treatment for EPP
* That there is an unmet medical need for a treatment for EPP
* That living with EPP is a tremendous burden
* How living with EPP affects your quality of life
It is important that you send your signed letter to the APF immediately.
Begin your letter:
Scott Gottlieb, M.D.
FDA Commissioner
U.S. Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD 20993
Dear FDA Commissioner:
Mail your letter to:
American Porphyria Foundation
4915 St. Elmo Avenue, Suite 105
Bethesda, Maryland 20814
Alternatively you can scan your signed letter and email it to porphyrus@porphyriafoundation.com.
All letters must be signed.
We will present all of the letters at once to the FDA!
Please send letters today or ASAP! Time is of the Essence!

Wednesday, June 27, 2018

Research is the key to your cure!


Research is the key to your cure! 


What is the most important element in research?

 YOU! 

You now have an opportunity to participate in a trial for a new EPP investigational drug. Contact the APF for details. 

YOU are our hope for the future. Contact the APF on 1-866-APF-3635 or email porphyus@porphyriafoundation.org.

An oral investigational drug has been developed with the potential to increase sunlight duration and tolerance in individuals with EPP. 


This phase II clinical trial is needed to understand if the investigational drug works and to ensure that it is safe. 

The study duration is 24 weeks including follow-up. You must be age 18-70 to participate and have a confirmed diagnosis of EPP. All travel is included and will be arranged by a concierge service.

"Remember...Research is the key to YOUR cure!”


Each Step Toward Finding an Effective Treatment is Important!

7209 GET INVOLVED STUDIES 7209 Effect of Oral Iron Therapy on Erythrocyte Protoporphyrin Levels in the Erythropoietic Protoporphyrias

Effect of Oral Iron Therapy on Erythrocyte Protoporphyrin Levels in the Erythropoietic Protoporphyrias


In order to participate in a study, you must personally contact 
the APF Edrin 1/866/APF/3635
For Diseases:
  • Erythropoietic Protoporphyria (EPP)
  • X-Linked Protoporphyria (XLP)

Background

In the medical literature, there are conflicting reports on whether iron improves symptoms in patients with EPP or XLP. Giving iron to people who are iron deficient is thought to improve EPP symptoms; however, this has never been systematically tested.
Therefore, the purpose of this study is to determine the effect of oral iron on EPP and XLP patients.
The research aims are:
  1. To determine if oral iron can decrease erythrocyte protoporphyrin levels in patients with EPP or XLP
  2. To assess whether oral iron can improve iron status and decrease plasma porphyrin levels and symptoms in patients with EPP or XLP
  3. To assess if there is an improvement in the quality of life in treated patients
This is a clinical trial, which means its purpose is to study an intervention or treatment. In this study all patients with EPP or XLP will be given a standard dose of iron pills and monitored for one year. There is currently no effective Food and Drug Administration (FDA) approved treatment for EPP or XLP in the US. Giving iron to patients with low ferritin (a measure of body iron stores) levels may help improve their EPP symptoms by decreasing erythrocyte protoporphyrin levels.
Participants will be asked come to the study site once every three months over the course of one year for a total of five visits. At these visits the study doctors will check in with the participant and some blood and urine samples will be taken. Participants will not be charged for any of the lab tests that are being done as a part of this study alone. In between each study visit, there will be one phone call to check in and see how each participant is doing. All patients in this study will receive iron pills at no cost to them.

Targeted Enrollment

To be eligible to participate, you must:
  • Be enrolled in the Longitudinal Study of the Porphyrias
  • Have a biochemical diagnosis of erythropoietic protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
  • Be at least 18 years old
  • Have a serum ferritin level of less than or equal to 30 ng/mL
You are not eligible to participate if you have:
  • Had a liver or bone marrow transplant, or have significant liver disease determined by the study doctor
  • A known or suspected allergy to oral iron
  • Used any other clinical or experimental therapy in the past 3 months
  • Another disease or condition that the study doctors judge would interfere with the study
In order to participate in a study, you must personally contact the

EPP Clinical Trial



You can be a medical hero! A new investigational drug for EPP is ready for clinical trial. All we need to begin is
YOU! The trial spots will fill up fast…join now! 

Contact the APF on 1-866-APF-2635 or email
porphyrus@porphyriafoundation.org to be connected to a study site. 


Don’t wait! Remember…Research is the
Key to Your Cure!


An oral investigational drug has been developed with the potential to increase sunlight duration and tolerance in
individuals with EPP. This phase II clinical trial is needed to understand if the investigational drug works and to
ensure that it is safe. The study duration is 24 weeks including follow-up. You must be age 18-70 to participate
and have a confirmed diagnosis of EPP. 

All travel is included and will be arranged by a concierge service.
"Remember...Research is the key to YOUR cure!"


Each Step Toward Finding an Effective Treatment is Important!


Monday, June 25, 2018

Porphyria Post IMPORTANT



Porphyria Post
Request
The New Drug Application for Scenesse has been submitted to the FDA.
Now is the time to work hard as patient advocates.
The APF is launching a new campaign to request Priority Review
Now is the time to send your letter requesting priority review for Scenesse. We need to move Scenesse, a long-awaited treatment for EPP, through the regulatory process as quickly as possible. Now that a complete new drug application has been submitted to the FDA - they have the option of granting priority review which moves the process faster. Please share with the FDA...
* That there is no treatment for EPP
* That there is an unmet medical need for a treatment for EPP
* That living with EPP is a tremendous burden
* How living with EPP affects your quality of life
It is important that you send your signed letter to the APF immediately.
Begin your letter:
Scott Gottlieb, M.D.
FDA Commissioner
U.S. Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD 20993
Dear FDA Commissioner:
Mail your letter to:
American Porphyria Foundation
4915 St. Elmo Avenue, Suite 105
Bethesda, Maryland 20814
Alternatively you can scan your signed letter and email it to porphyrus@porphyriafoundation.com. 
All letters must be signed.
We will present all of the letters at once to the FDA!
Please send letters today or ASAP! Time is of the Essence!
Update your records....
We have moved.
The American Porphyria Foundation is excited to announce out move to Bethesda, Maryland - next door to our nation's capital. Please update your records with our new address.
American Porphyria Foundatin
4915 St. Elmo Ave., Suite 105
Bethesda, MD 20814
Telephone: 301.347.7166
porphyrus@porphyria.com
Rare Disease Legislative Advocates is hosting In-District Lobby Days to facilitate meetings for rare disease advocates across the country with members of Congress in their local offices during the 2018 summer in-district work period (July 31st through Sept. 4th).
Meeting with your representative and senators throughout the year is critical to building key relationships. These meetings also provide an opportunity to discuss legislation that is meaningful to the rare disease community, and to highlight the importance of the Rare Disease Congressional Caucus. Make YOUR voice heard as you advocate on behalf of the rare disease community!
Registration for In-District Lobby Days is FREE and open through July 4th. You will be provided the option to specify you availability and the distance you are willing to travel for a meeting.
All In-District Lobby Day participants will be invited to a webinar on Thursday, July 26th,at 2pm EST to go over key legislative issues and tips for successful meetings. Legislative asks will be available RDLA's website by late July.
Get Involved
• Research •
 Donate 
• Visit our Website •
Contact Information
Is your contact Information up to date?
If not please give us a call @ 866-APF-3635 or Email to porphyrus@porphyriafoundation.com
American Porphyria Foundation| 1.866.APF.3635 | porphyriafoundation.org
STAY CONNECTED

"Remember....Research is the Key to Your Cure!"

The Porphyria story of Victor LaFae with HCP

Porphyria story - HCP - Victor LaFae I’m told that I was a typical happy baby for the first few months of my life. I reached all my mile...