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EPP Dad
Posted on: Mar 15 2020, 08:59 AM


Group: Member
Posts: 7

Trial is scheduled to start in April not August.
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EPP Dad
Posted on: Apr 2 2015, 05:26 AM


Group: Member
Posts: 7

From APF Facebook page:

Desiree Lyon NBC dateline will show in late April. Trying to be near awareness week....
They have filmed scenesse ,too, which I believe will be a big help getting it here
March 30 at 10:58pm · Like · 1
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EPP Dad
Posted on: Feb 25 2015, 03:30 AM


Group: Member
Posts: 7

Yes he did. He stayed out in the direct sunlight until it was uncomfortable. A couple of the more obvious signs that he would have received if he had the real drug Scenesse would be getting some freckles and a tan which he never got. He knew he had the placebo within about 5 days of the initial implant.
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EPP Dad
Posted on: Feb 24 2015, 12:14 PM


Group: Member
Posts: 7

Reality,
The severity of EPP varies between individuals. My son for example can be in direct sunlight for approx 20 min before he starts to feel a reaction. At that time if he would get out of the sun he probably would be ok. If he remains in the direct sunlight for an extended period---say 40 min he could have a full blown reaction that takes days to recover. Generally on him there are no signs of any scaring, rashes etc. he looks normal but he says it feels like his face, hands and feet ( if they are all exposed) are in a scalding bucket of water. The only relief at this point is to stay out of the sun for days until the pain subsides. Sunscreens, beta carotene, other sun lotions do not work.

My son participated in the phase III EPP trials---unfortunately he received the placebo. He spoke with a number of people who got the "real drug Scenesse" in both Phase II and Phase III and each one of them said it was truly a life changing experience. They were able to stay outside in the sun for 6-8 hrs before feeling any type of reaction ---the reaction was short lived and they could go on with everyday living. One person said that he was able to go on a beach vacation for the first time ever with his family and was able to be in the sun for most of the day. Staying outside this long was probably a combination of both indirect and direct sunlight I'm assuming. As part of the clinical trial my son had appt's at the participating hospital every 60 days or so to get a new implant. Part of the visit included various tests----blood tests, general health tests and a light test where they measured the tolerance to a heated lamp/light that they shined on his hands and his lower back----I guess to simulate sunlight. In his case he could only tolerate a few minutes---I can't remember exactly how long but the people who received Scenesse were able to tolerate the light for a much longer time versus what they could tolerate before the drug. Maybe this is where the extra 7 minutes came from---I don't know. I know for my son any extra time in the sun would be a bonus. Anyway I hope I answered your question in a long round about way.



Thank you for the kind wishes---best of wishes to your family as well.
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EPP Dad
Posted on: Feb 24 2015, 12:17 AM


Group: Member
Posts: 7

Forgot to mention ---they will also be interviewing EPP patients and the benefits of Scenesse --before and after taking the drug.
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EPP Dad
Posted on: Feb 23 2015, 11:43 PM


Group: Member
Posts: 7

This is an excerpt from the APF Facebook page---from Desiree Lyons


FYI. To let you all know what the APF has done for rare disease week and Awareness Week. we have secured three television programs on porphyria, NBC Dateline, already filmed and ready to air, Discovery Channels new program Diagnosis Unknown, which has been filmed and to be aired. tba. And a PBS special to be filmed soon. The entire program will be about porphyria, an exciting endeavor. We are selecting patients for the program with the production team now. It will be primarily about acute porphyrias.
national radio Interviews to be aired to over 50 million people . They are interviewing two AiP patients ,
Announcements on fb groups , in three ENews, and News sections.
national radio shows with expert to be aired to appx 50 mil. Last year dr Joseph Bloomer did six hours of interviews aired to sim number for rare disease day and porph awareness week. We hope the timing will be same. We are grateful to him and other experts who devote their time voluntarily for us. he spent his vacation week doing these programs. Tony will be doing a radio show. I have done many. Everyone can call your local stations and secure an interview.
Plus, we have joined with 124 rare disease organizations together to promote rare diseases in general ...NORD. We have united to promote rare disease day as we did to get Orphan Drug Act approved . Followed closely behind is porph awareness week.
Working and making headway for rare diseases in the efforts for 21st Cure Act legislation , which will benefit all rare diseases. Joining other groups for Capitol Hill visits. Ppl who live in the DC area will be attending representing APF / porph . Contact the APF if you want to participate.
Produced 3000 materials /Awareness Packets for rare disease day feb 28 and Porphyria Awareness Week ,April 11-18 for porphyria ppl to enhance awareness in their communities . And fact sheets by tens thousands . Distributing the DVD Porphyria live by the thousands, including to porph community who want one .
And
Our Care About Rare gathering in Houston, RDD 2-4. RSVP to join .
Would love to hear what others are doing.
And
THE SHADOW RIDE in Oklahoma.
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EPP Dad
Posted on: Dec 3 2014, 09:39 AM


Group: Member
Posts: 7

I am a first time poster here on this forum. My son has EPP and participated in clinical trials for Scenesse. Although he received the placebo he spoke with other people who received the real drug. Each one of them said that Scenesse was truly "life changing". I am very proud of my son for sticking it out because it would be real easy to quit after receiving the placebo but he knew it would be for the greater good. After participating in conf calls with the APF last summer we were given hope and very strong inklings that the drug would be eventually approved in Europe and that the FDA would fast track the drug. After the announcement in late October that the EMA had approved the drug I contacted the APF and they plan on having additional conf calls to update everyone on the FDA approval process. I anticipate this will happen early next year after Europe officially ratifies Scenesse. It is my gut feeling that they would like to have FDA approval very soon so that EPP sufferers would have access to the drug before the summer of 2015. For the record I believe so much in this drug that I did purchase shares (small scale) CLVLY here in the US.
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