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Showing posts from February, 2017

Update: Acute Porphyria FDA Meeting March 1st!

Update: Acute Porphyria FDA Meeting March 1st!Based on the agenda, we will be welcoming live questions at designated points during the meeting via live chat. Please comment/provide your questions in the chat section on the webcast. If you are unable to provide question(s) there, please email your question(s) to Jessica at
Jessica@porphyriafoundation.org. To view the meeting live, we will have a webcast streaming in real time. Here is the link: https://youtu.be/AX20OvotxHA We have attached the link for the PFDD Meeting Agenda for your re-view. Here is the link:http://www.porphyriafoundation.com/…/PFDD-Meeting-Agenda.pdf If you are planning to speak at the FDA Meeting, please submit your speech to the APF TODAY to lyonapf@aol.com. You are welcome to say whatever you want, however, we need everyone’s story sent in order to know where to place you within the program. Contact us with any questions! We can't wait to see everyone!! "Remember....Research is the key to your cure!"

PFDD Meeting Agenda

We are only DAYS away!We have attached the link for the PFDD Meeting Agendafor your review. Here is the link: http://www.porphyriafoundation.com/sites/default/files/PFDD-Meeting-Agenda.pdfIf you are planning to speak at the FDA Meeting, please submit your speech to the APF  TODAY to lyonapf@aol.com, so that we can know where to place you within the program!!We can't wait to see everyone!!

FDA Meeting Update please note

For those of you attending the FDA Meeting, if you haven't registered, please do so here: https://www.eventbrite.com/e/patient-focused-drug-development-meeting-acute-porphyrias-registration-31068098519If you are planning to speak at the FDA Meeting, please submit your speech to the APF by FRIDAY, so that we can know where to place you within the program!!The meeting will be webcast live on our YouTube Channel. Here is the link:https://www.youtube.com/watch?v=AX20OvotxHA**For those of you not attending the meeting in person, you do nothave to register in order to watch the live webcast. **If you misplace this link, you can also gain access via the APF website.   Click on the "Register Now" link on the homepage of the APF website under the FDA announcement and it will bring you right to it!   Note:  The chat feature will only be accessible through YouTube Live! 
The APF meet and greet will be on February 28th at the College Park Marriott (same place as FDA meeting the next …

Rare Disease Week on Capitol Hill: February 27, 2017 – March 2, 2017

Want your voice heard? Submit your story TODAY!Rare Disease Week on Capitol Hill: February 27, 2017 – March 2, 2017Rare Disease Week on Capitol Hill brings rare disease community members from across the country together to be educated on federal legislative issues, meet other advocates, and share their unique stories with legislators. Submit your stories TODAY to let Congress know about your experiences with this rare disease; how it has impacted your life and let them know which issues are most important to you.You can submit your personal perspective http://rareadvocates.org/rdw-old/patientstories/ to be hand-delivered to your Representative and Senators on Lobby Day.
The deadline to submit perspectives is Sunday, February 19th. The registration to attend in person in closed, however, you can participate remotely by watching the livestream of the Legislative Conference on Tuesday, February 28th, from 9am to 5pm ET. To register for the free live stream register here: http://action.eve…

Clinuvel Pharmaceuticals Ltd

The February 2017 edition of CLINUVEL's newsletter is out today. CEO Dr Philippe Wolgen provides latest company updates and information for 2017.Clinuvel Pharmaceuticals LtdFebruary 16 at 4:03am The February 2017 edition of CLINUVEL's newsletter is out today. Our CEO Dr Philippe Wolgen provides latest company updates and information for 2017.
file:///C:/Users/Bamy/Downloads/20170216Newsletter.pdfCLINUVEL Newsletter - February 2017 - Clinuvel Pharmaceuticals CLINUVEL.COM

FDA Meeting Meet and Greet and Webcast Link!

FDA Meeting Meet and Greet and Webcast Link!The webcast link is ready!  Here is the link:https://www.youtube.com/watch?v=AX20OvotxHAIf you misplace this link, you can also gain access via the APF website.   Click on the "Register Now" link on the homepage of the APF website under the FDA announcement and it will bring you right to it!   Note:  The chat feature will only be accessible through YouTube Live! The APF meet and greet will be on February 28th at the College Park Marriott (same place as FDA meeting the next morning).  The room will be open for an informal meeting space from 5-6PM and there will be a mandatory preparation meeting for all attendees from 6-7PM.  After this meeting, you are welcome to go to dinner, hang out with friends, etc.  The hotel has an on-site restaurant, The Common, and advance reservations are recommended.  Please let the APF know if you plan on attending the meet and greet and preparation meeting.  We need to make sure the space is large enou…

Acute Porphyrias By By Herbert L. Bonkovsky, MD, University of North Carolina at Chapel Hill, and University of Connecticut Health Sciences Center ; Vinaya Maddukuri, MD, Carolinas Medical Center

Acute Porphyrias By Herbert L. Bonkovsky, MD, University of North Carolina at Chapel Hill, and University of Connecticut Health Sciences Center;Vinaya Maddukuri, MD, Carolinas Medical CenterCLICK HERE FOR
Patient Education
NOTE: This is the Professional Version. CONSUMERS: Click here for the Consumer VersionPorphyriasOverview of Porphyrias Acute PorphyriasOverview of Cutaneous PorphyriasPorphyria Cutanea TardaErythropoietic Protoporphyria and X-linked ProtoporphyriaAcute porphyrias result from deficiency of certain enzymes in the heme biosynthetic pathway, resulting in accumulation of heme precursors that cause intermittent attacks of abdominal pain and neurologic symptoms. Attacks are precipitated by certain drugs and other factors. Diagnosis is based on elevated levels of the porphyrin precursors δ-aminolevulinic acid and porphobilinogen in the urine during attacks. Attacks are treated with glucose or, if more severe, IV heme. Symptomatic treatment, including analgesia, is given as ne…