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FARAFARA Cure FA

FARA Ambassador Program

The FARA Ambassadors is a united team of patient volunteers living with FA who are committed to supporting FARA in the search for a treatment and cure.

Together we seek to know more about FA, and FARA so we can be prepared to represent the community when the opportunity arises; speaking at events, to volunteers, potential donors, scientific groups, pharma partners, media interviews and other awareness and fundraising opportunities. We believe support is key to continued success toward our ultimate goal of treatment and a cure. Participants in the FARA Ambassador Program are passionate about building and upholding relationships within the FA community.

FARA Ambassador Program Mission Statement:

The FARA Ambassadors are positive, supportive, peer representatives for the FA community, actively raising awareness and funds for FARA. To learn more about the FARA Ambassador Program or to have a FARA Ambassador speak at your event, please contact: info@cureFA.org.

The Ambassador Blog

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This blog is a vehicle for the voice of the FARA Ambassador Program and features posts from Program participants and friends from the FA community on a wide range of topics.

The blog features posts about FARA events, articles about living with FA, spotlights on individuals throughout the FA community, weekly interviews to “Meet the Community,” and more. We hope that you will be inspired, uplifted and encouraged through the FARA Ambassador Blog!


 

FA Patient Focused Drug Development Meeting - Your opportunity to tell the FDA what is important to you in finding a treatment for Friedreich’s Ataxia

Drug Development Meeting 4

What is a Patient Focused Drug Development Meeting?

In 2015 the Food and Drug Administration (FDA) began a new initiative to expand the way it looks at therapies, specifically seeking input from patients. The agency wanted to hear directly from the community about patients’ needs, rather than assuming that internal FDA scientists understood every disease and patient population. Thus, it launched the Patient-Focused Drug Development (PFDD) Initiative, a series of public meetings led by the FDA and designed to systematically gather input from patients regarding their experience of living with specific diseases, the preferred impact of potential treatments, and the benefit/risk analysis when considering a new targeted medication.. The FDA scheduled the initial 20 meetings, many of which have taken place and are considered very successful. In order to reduce their administrative burden however, the FDA, has now moved to a new model where such meetings are led by patients and patient groups, with the FDA attending and participating. FA has been selected to be one of the disease areas to host such a meeting in 2017.

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Patient Perspective: Frankie's Experience Participating in a Clinical Trial

PatientPerspective Frankie1Hello! My name is Frankie Perazzola, 24 years old and diagnosed with FA at age 22. I started STEADFAST/Actimmune/ Interferon Gamma in February 2016 which later came to an unexpected stop in November of 2016. This drug was supposed to increase levels of frataxin which all FA patients lack. Since frataxin is essential for energy production, Actimmune was expected to increase the specific protein which would potentially increase energy levels and possibly increase mitochondrial function. Two simultaneous shots, every other day were required by injection in my arms and legs. The first portion of the study, I did not know if I was injecting the drug or a placebo. However, I was guaranteed the drug for the second portion and even had the option of continuing until approval by the FDA- which I later opted for. 

Another option I went for was the muscle biopsies- which is essentially a mini operation requiring a small incision on the thigh that leaves you a tad sore afterwards. No anesthesia required. I learned through my main neurologist, Dr. Perlman, that muscle biopsies were a better way to measure frataxin levels. By better, I mean a more accurate way to measure the levels compared to just giving blood and cheek swabs, because muscle is considered affected tissue. If I was participating in a trial, I wanted to give everything I could in order for my results to be as precise as possible. 

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FA Hangouts - February 2017

FebHangoutsThe FARA Ambassador Program presents an FA Hangout talking about: romantic relationships and FA. Are you in or looking for a romantic relationship? How has, or hasn’t, FA affected that? How do you keep your relationship going strong despite your chronic illness, FA?

At FA hangouts: we laugh, we connect, we hangout, we learn from and about each other...it is so fun! It is just an informal discussion with a theme. We just talk when the theme has run its course, no pressure. People who just want to listen are very welcome!

Every hangout is open to anyone with FA and a translator if needed for hearing or speech difficulties.

When: Hangouts will be held Tuesday February 21st, Wednesday February 22nd, and Thursday February 23rd (mark your calendars, FA Hangouts will be the last Tuesday, Wednesday, and Thursday of the month, except on holidays and the like) at 8-9:30 PM EST all three days.

How: Email fahangouts@gmail.com to sign up or ask questions. Google (not us!) limits hangout capacity to 10 devices. FA Hangouts do fill up. It is first come, first served. So, the earlier you sign up, the more likely you are to get the date and time you want. Lately all our hangouts have been filling up. If you signed up and can’t make it, please let us know so we can give the slot to someone else.  Add a comment

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Patient Perspective: Michael's Experience Participating in a Clinical Trial

MichaelGehr1On January 19, 2016, I was diagnosed with FA. My family and I decided to take an educated approach, and we did just that. Within a few weeks, we had read countless websites about the symptoms and what we could expect with my new diagnosis. Soon, we began learning about the science behind FA, and we eventually found ourselves reading about clinical trials. By April, I was signed up for one; my first visit was to be on the 15th.

Even though it had been less than 3 months since my diagnosis, I knew that a clinical trial was something I wanted to involve myself in. It was something that just made sense for me. Added bonus: The drug company, Reata, was able to pay for plane tickets, meals, etc.! I knew that as well as being helpful for the community and everyone looking for a cure for FA, I may be able to benefit also. For example, one of the tests I did was a stress test, where progression is measured by viewing one’s exercise capacity via a recumbent bicycle. Largely because of this test, my family purchased an exercise bike which I use daily. As another example, since I am in highschool, I knew that this trip would be a learning experience for me. I was able to learn a lot about restrictions and necessary testing for a clinical trial. Every doctor was able to teach me about their specific test, and the lead doctor, Dr. Hoyle, taught me about basic neurologic tests, and even some about myself. 

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A Mom's Perspective on Participating in Clinical Research

Six months ago, our 10-year-old son Jake was diagnosed with Friedreich’s Ataxia. When his neurologist explained FA, she told us that FA has no treatment and no cure. The diagnosis is staggering. Learning that there is no treatment and no cure (yet!) took our breath away.

JakeJuip10After taking a few months to cope privately with the diagnosis, we were ready to talk about it publicly. By that time, we had combed the internet and had read everything we could find about FA. We are a family always on the go. We just couldn’t sit idly by and watch the disease progress. We needed to do something to respond to Jake’s diagnosis. Consequently, we were so grateful to learn about and attend the Patient Symposium in Philadelphia last October. At the symposium, we learned about biomarker studies and clinical trials. (Biomarker studies have no direct benefit for the participants, but the studies gather specific information about FA patients that can be used in future clinical trials. Clinical trials explore whether or not a specific therapy is safe and effective for humans.) Each study and trial has specific qualification criteria, so I compiled a list of the studies and trials that Jake was eligible for, and I called Jen Farmer, Executive Director of FARA. She’s so knowledgeable and helpful. She spent time explaining each of them and their objectives. Importantly, she also outlined the time commitment for each. Because FA is a progressive disease, most of the studies and trials require repeat visits to track the disease’s progression over time. All of the studies and trials would require travel, so we needed to balance our desire to contribute to FA research versus the practical constraints of school schedules, childcare for our other children while we travel, and cost. 

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