Skip to main content


Showing posts from October, 2016

Clinuvel Information

CLINUVEL PHARMACEUTICALS LTD[ASX: CUV; Nasdaq International Designation ADR: CLVLY; Xetra-DAX: UR9] today announced that it will meet with the US Food and Drug Administration (FDA) on 7 November to formally discuss lodging its new drug application (NDA) for the novel drug SCENESSE® (afamelanotide 16mg). The pre-NDA meeting will focus on finalising requirements for filing SCENESSE® with the FDA for the treatment of adult patients with the rare genetic disorder erythropoietic protoporphyria (EPP). 

“This is the last step required of CLINUVEL before we ask the FDA to review the risk-benefit profile of SCENESSE® in a submitted dossier,” CLINUVEL’s Acting Chief Scientific Officer, Dr Dennis Wright said. “A successful risk-benefit review will enable CLINUVEL to make the drug available to US EPP patients.”

The pre-NDA meeting, with the FDA’s Division of Dermatology and Dental Products (DDDP), is part of ongoing dialogue between CLINUVEL and the FDA since CLINUVEL commenced a clinical program…

Accused of Being a Hypochondriac, Lisa is Finally Diagnosed with Acute Intermittent Porphyria- Global Genes

Accused of Being a Hypochondriac, Lisa is Finally Diagnosed with Acute Intermittent Porphyria
A swollen stomach made her look pregnant, but the pain — “burning,” “shocking,” “horrible,” “out-of-this world,” as she describes it — was worse than childbirth, leaving her doubled over and gasping for breath. Symptoms came and went, seemingly at random. Vomiting. Constipation. High blood pressure. A racing pulse. Numbness in her hands. Paralysis in her right foot. Lisa Kehrberg spent several days in a hospital, but every test came back normal. And the doctors began to wonder if perhaps she had been under a lot of stress lately.

Important Things To Know Before The FDA Meeting For EPP On MONDAY

Important Things To Know Before The FDA Meeting For EPP On MONDAY
The FDA Meeting for EPP is just around the corner! The FDA has distributed additional information for all registered attendees. The check in process is lengthy, so everyone needs to be sure to arrive to the FDA at 9AM on Monday, October 24th. As a reminder, the APF will have a shuttle bus available to transport attendees between the Holiday Inn – College Park and the FDA. Transportation: If you are taking a cab, ask the driver to drop you off at Building 1. If you will be driving and need to park, there will be signs labeled “Event Parking” pointing you in the direction of various surface parking lots (Southeast, 132A and 132B). Parking spots may be difficult to secure that will be close to Building 1, but there will be shuttles circulating to all parking lot bus stops every 5 minutes that can pick you up. Make sure to tell the shuttle driver to drop you off at Building 1. Here is FDA’s transportation page for more inform…

The FDA Meeting for EPP is Less Than 3 Weeks Away!

The FDA Meeting for EPP is Less Than 3 Weeks Away! The APF is busy preparing for the upcoming EPP meeting at the FDA on October 24th, 2016.  We are very excited about the large number of attendees and hope even more people RSVP to us in the coming weeks. When registering for the FDA meeting, there is an option to indicate if you are interested in participating in a panel.  If you have signed up to participate in the panel during the meeting, you should have received an email with a list of discussion questions from Meghana Chalasani (FDA meeting coordinator), in addition to your registration confirmation email.  These questions must be answered and returned to Ms. Chalasani.
The deadline to submit your answers for the panel has expired.
You will be notified of your panel status at least 1 week in advance of the meeting.  If you have signed up to speak at the panel, but did not receive the follow up email from Ms. Chalasani, then you are not being considered for the panel.  You must contac…

Taking Notes and Recording Your Visit

Taking Notes and Recording Your Visit~

Have you ever felt overwhelmed during a Doctor's appointment in which you received a large amount of information?  I know I have.  Perhaps you didn't ask any questions at the time even though you felt you had questions once you had time to process all of the details. 

     Your not alone.  Many patients find themselves with many unanswered questions once they have a chance to look over the printed materials and digest the information.

These tips may help you feel more informed during your next visit:

Write down a list of specific questions.
Bring someone to the appointment with you to listen, take notes, and record the visit.
Verbally summarize the instructions for the doctor or the nurse
Ask pointed questions such as: "can you please review with me again what I need to know about getting Porphyria testing done right?"

Many health care providers are using a technique called "Teach-Back" to ensure you understand what they just …

Your Stories

YOUR STORIES- Are you a porphyria survivor or know someone with an inspiring story to tell? How has porphyria affected you or your loved ones? Whether it's a story, a poem, a picture, fundraising event, a picture or a painting, share YOUR story in your way. Your story will be made available in a published blog and the upcoming 2017 Porphyria Awareness Week. Please email me your type & name with what you want to share

A journey to successful protection with Scenesse. #SuccessWithScenesse

A journey to successful protection with Scenesse.
Our son, JT von Seggern has Erythropoietic Protoporphyria, EPP. EPP negatively effects the liver, results in vitamin D deficiencies and causes excruciating pain – all from exposure to the sun. JT has experienced the painful effects of EPP since he was 2 years old. It took us 9 years of extensive research and numerous doctors’ visits, including a trip to Johns Hopkins to finally get a diagnosis for what was causing our child so much pain. While gaining a diagnosis was a huge relief, learning that there were no treatments and no cure for EPP quickly knocked the air out of our initial relief. Exactly how does one go about protecting their now 11 year old, sports loving son, from exposure to the sun?
Through the ensuing years, JT experienced frequent pain, swelling, scarring, missed school days, and numerous rounds of Prednisone to bring down the swelling caused from exposure to the sun. Flash forward to the spri…