friedreich's ataxia scientific symposium 2019

We Interrupt the Regularly-Scheduled Program for a Special Announcement…

Hi, friends. How’s your week been? Mine’s been really fun and a little exhausting. I travelled from NC to PA to attend rideATAXIA Philly 2019 and the 12th annual scientific symposium hosted by FARA (Friedreich’s Ataxia Research Alliance).

The ride is a fundraiser for FARA that takes place in different locations across the US every year. Right now, I’m reading a memoir, Shifting into High Gear, by Kyle Bryant, the founder of rideATAXIA. He discusses the shame and self-loathing and despair with which he grappled after being diagnosed with Friedreich’s Ataxia; he had always loved sports, and now, his future had been ripped away from him, and his abilities were deteriorating. Then…he discovered three-wheel trikes, and the rest is history. I’m eager to read of his trek ACROSS THE US on a trike. Amazing stuff is possible with some courage and ambition, y’all. Anyway, I’ll definitely post a review of that here (and buy it for several loved ones for Christmas, hehe). At rideATAXIA Philly 2019, we raised over $500,000 for FARA!

The conference was great also, but I will be honest and admit that I didn’t go into the conference feeling as hopeful this year. I’ve been told that a treatment or cure is “just on the horizon” for years now, so I was starting to feel jaded, wondering, “so…is ‘the horizon’ ever going to get here?”

We learned about ongoing clinical drug trials and new research throughout the day, and after several hours, I wondered if perhaps we could leave early; the last session is a patient panel, so it’s not crucial. But thank the Lord we stayed the whole time. As everything was wrapping up for the day, Ron Bartek, the founder of FARA, stepped to the mic to share a press release.

He tearfully read the following headline–“Reata Announces Positive Topline Results from the MOXIe Registrational Trial of Omaveloxolone in Patients with Friedreichโ€™s Ataxia”

As he read the press release, choking on his words every few sentences, tears stung my own eyes and cheers erupted throughout the room. It was a wonderful, intimate, unforgettable moment for the FA’ers and their families in the room.

The FARA treatment pipeline as of June 2019

Though several clinical drug trials have been conducted in search of a treatment/cure, no trial has ever successfully met its endpoint. That statement is no longer true. Since this treatment was successfully shown to help FA patients, Reata Pharmaceuticals is now pursuing FDA-approval, and the medicine will be available for patients at some point in the (hopefully) near future. [Pricing and insurance will be a bear, but I’m not letting that rain on my parade.]

Of course, a treatment is far from a cure, so our efforts cannot slow down (and I can’t buy a pair of high heels just yet). But what we need while we wait for a cure, which is the enemy of all progressive diseases, is TIME; I hope this new medicine will halt or at least slow down disease progression until gene therapy is developed to cure my condition and, incidentally, many others that are rooted in a person’s DNA. Sidenote, it’s crazy to think that DNA’s structure was discovered in the 1950’s, and look how far we’ve come already. Science is awesome!

My usual readers know I’m a Christian, so maybe my last statement seems misaligned, but I don’t subscribe to the notion that science and faith are enemies. When I look at the stars in the sky on a clear night or consider how many grains of sand comprise a shoreline, I feel awed by our creator’s imagination; maybe the fact that we have DNA, unique instructions for our bodies/lives, just goes to show how we are “fearfully and wonderfully made.” Here is a statement I included with another social media post: For Lord knows how long, I have prayed, “Guide the hands and minds of the doctors and scientists in FARA to find a treatment or cure.” I don’t want to take anything away from the hard work of so many, but I do believe that God is working things to the good…in higher ways I can’t fully understand or even articulate. My heart was already full of joy and hope, but it feels like my heart grew a little bigger. Thankful to God for the people and events that led to this point and eager to see what the future holds.ย โ™ฅ

I’ll be spending the next few days catching up with my fellow bloggers and replying to comments. BTW, thank you to those who left thorough and thoughtful comments on last week’s post; you guys teach me so much.

Until next time, thanks for reading!

21 comments

  1. I am so thankful for this day and the wonderful news.I am glad that I have had opportunity to attend this Symposium in the past so I can relate to sitting all day with barely a glimmer of hope. I am glad that Ron Bartek got to give the Press Release as he and Rachel have worked tirelessly for all of you with FA. I will continue to pray for all of you and the studies and clinical trials that continue..For happier days ahead.

    Liked by 1 person

  2. Oh what wonderful news! I am so Thankful our Prayers are being answered. God is GREAT! God is an awesome God! I will continue my prayers Lily for you all with this disease that you get this medication ASAP! I’m sure the money raised will surly help to speed things along, I pray so anyway.. Love you Deb

    Liked by 1 person

  3. So happy for you for the good news! I love your ideas on science! The etymology of the word science means “what is known, knowledge (of something) acquired by study, …” https://www.etymonline.com/word/science#etymonline_v_22918
    There are plenty of Christians who are scientists! I believe God wants us to explore and discover the intricacies of His creation! The more awesome we find it to be, the more we can realize how awesome HE is!

    Liked by 1 person

  4. Awesome news!! ๐Ÿ™๐Ÿ™๐Ÿ™ I pray that this treatment leads to a real cure. And the burgeoning field of epigenetics (not even sure if that is what it is called??) is so intriguing to me (confusing too, but I agree with you — the science that is discovering all these new things about our DNA is not opposed to God IMO it is another reminder of God’s intricate and infinite glory!!)

    Liked by 2 people

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