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Showing posts from November, 2019


The American Porphyria Foundation is pleased to announce that the Food and Drug Administration has granted APPROVAL for GIVLAARI (Givosiran) for the treatment of Acute Hepatic Porphyria! US patients will now have access to this preventative RNAi treatment. This approval is the first step in gaining access to GIVLAARI (Givosiran) in the US. Availability to patients will progress as Alnylam Pharmaceuticals pivots from FDA approval to commercial availability.
The APF will update you frequently on progress related to access and distribution of GIVLAARI (Givosiran) treatment.
Congratulations to YOU, our patient community, who participated in the pivotal Phase 2 and Phase 3 clinical trials that led to this approval. You are our medical heroes!
Click Here to See the Full FDA Press Release Link: FDA approves fi…

Protect the Future

Protect the Future
The Protect the Future campaign was established to attract and train the next generation of doctors and specialists in the field of Porphyria. Over the next decade, we will lose ninety percent of our valued Porphyria experts. These men and women have led Porphyria research, testing and treatment for the past 30 years. Without financial support, we run the risk of losing knowledge of the disease, quality testing, diagnosis, and treatment, and ultimately a cure. Protect the Future supports young doctors who work and study with long-time experts, seeing patients and doing research, in order to gain the expertise they will need to care for the U.S. Porphyria patient population for decades to come. Protect the Future is a separate fundraising campaign with its own specific needs. We are seeking additional donations for the specific purpose of locating and training new Porphyria specialists. You and I are pulled in so many directions for our donations, such as churches, poli…

Where can I find out how to cope with an undiagnosed condition?

Trying to find an underlying diagnosis for many conditions can be a very long and frustrating experience. With more rare conditions, a diagnosis can often take many years. Although this can be incredibly difficult, the following information may help you navigate through the process of trying to obtain a diagnosis. If YOU suspect you have one of the Porphyrias please contact the American Porphyria Foundation 1.866.APF.3635 or visit

Where can I find out how to cope with an undiagnosed condition?To learn more about how to deal with genetic or rare conditions that have no definitive diagnosis, see: Learning about an Undiagnosed Condition in a ChildLearning about an Undiagnosed Condition in an Adult
Global Genes has developed a resource for people with undiagnosed conditions called "Becoming An Empowered Patient: A Toolkit For The Undiagnosed."

Are there research programs available for people without a diagnosis?
Yes. If an individual’s health care providers an…


IMPORTANT UPDATE November 11, 2019 Mitsubishi Phase 2 Clinical Trial Meets Endpoint for Treatment of Erythropoietic Protoporphyria (EPP) Mitsubishi Tanabe Pharma Development America, Inc. today announced successful completion of the Phase 2 clinical trial of MT-7117, an investigational oral treatment under development for the prevention of phototoxicity (including severe pain on exposure to sunlight) in patients with Erythropoietic Protoporphyria (EPP). In this Phase 2 proof of concept trial, MT-7117 met its primary endpoint and was generally well tolerated with an acceptable safety profile. "The results of ENDEAVOR are very encouraging and will pave the way for a pivotal trial to evaluate the safety, efficacy and effectiveness of MT-7117 as an oral once a day treatment option for EPP” said Robert Desnick, MD PhD, Dean for Genetic and Genomic Medicine, Icahn School of Medicine at Mount Sinai, NY and Lead Investigator o…


New APF items are in the store! check it out!

Todays Porphyria Post All items, prices and sizes are on the website. These include tax & shipping. If you have any questions regarding placing an order please email Gift giving is just around the corner show your loved one you support them. Did you know that 100% of your purchase goes right back to the APF? You can find the APF store here: — at American Porphyria Foundation.


Porphyria Post Have You Joined Our Open & Closed Facebook Groups? Porphyria Research Group? Have you checked out the APF Facebook groups recently? Did you know APF has seven Facebook groups? We moderate one public group along with six private disease-specific groups that you can request to join. The communities are very active and it is a great way to connect with other porphyria patients and caregivers. In addition, the APF regularly posts publications, research, updates and other educational information As an ultra-rare condition it can seem like you are alone but YOU ARE NOT! ….come join us! Below are the Official 7 APF Facebook Groups Public Group
Porphyria - American Porphyria Foundation Private Groups
APF: Porphyria - AIP, HCP, VP APF: Porphyria - PCT APF: Porphyria - EPP APF: Porphria - CEP Porphyria International Support & Education Group Porphyria Research SCENESSE® In Australia The Australian Therapeutic Goods Administration (TGA) has enabled Priority Registration Pat…

Medical Moments

MEDICAL MOMENT Below are the most prevalent symptoms of acute porphyrias (AIP, VP, HCP, ADP): abdominal pain and vomiting constipationpain in the back, arms and legsmuscle weakness (due to effects on nerves supplying the muscles)urinary retentionpalpitations (due to a rapid heart rate and often accompanied by increased blood pressure)confusion, hallucinations and seizuresinsomnia Sometimes the level of salt (sodium and chloride) in the blood decreases markedly and contributes to some of these symptoms. See the APF website: