Shandra

The beach is my favorite place to be. Yeah it’s extremely difficult and exhausting to get on and off of it (because i am a person that still walks but cannot independently on sand) and the beaches where I am mostly have stairs and no accessibility. I do a lot of butt scooting and if I’m with someone I hang onto them for dear life lol. But that…[Read more]

My iPhone flashlight is my very best friend. I have a walker but that really doesn’t make a difference, I NEED to see

The last time I ran decently enough to call it running was 10 years ago, and I remember I was filming a “How to Be a Ninja” video for YouTube because that’s what all the cool preteens were doing (lol). The last time I moved my body at a pace quicker than walking but in a manner that looked really worrisome and not really resembling of running was…[Read more]

Hahahahaha! I don’t think my legs are long enough bc I’ve never had this problem. I always clip my ankles. Its like when we had razor scooters, but far less enjoyable.

It’s definitely speeding up. 🙂

I 100% don’t think it’s something to put all hope in. There are much more exciting things in development 🙂 but this might end up being a drug that works alongside another drug to alleviate some of FA’s progression (synergistic benefits). That would be exciting while we wait for something that really targets the root of the problem. And since we…[Read more]

Matt that’s so cool omg. Like, REALLY cool. 🙂

Have you tried contacting the study team? I’m positive they could give you proof of your FA. They probably still have some of your blood in a -80 freezer lol.

Technology has definitely improved though and therefore if you just did a new test now they could actually give you your repeat numbers…[Read more]

I doubt they have data on this that is shareable at this point. If animal studies have been initiated then I suspect any suggestive data is too preliminary and it’d therefore be irresponsible for them to share.

The in vitro studies suggest a dose-dependent increase in Frataxin and the patent gives them the rights to test up to 600 mg daily in…[Read more]

Basically, the research group that discovered Etravirine’s potential has filed for a patent giving them the rights to develop/test this as a treatment in FA patients.

Since etravirine is already a drug on the market, it is claimed by someone for use in HIV. In FA, no one owns Etravirine, so theoretically any company could start clinical trials…[Read more]

I’m so excited about this. Great things come from collaborations and easier access to data. This is basically a pipeline for FA researchers everywhere. 😀

Based on their presentation at CHOP in October, I’d say UF is the farthest along. And based on press release it seems like Votager isn’t too far behind. I know nothing about the other companies, beyond the fact that they have an FA gene therapy project.

The Moxie trial is not gene therapy. 🙂
I really like the way FARA breaks down drugs into categories in their treatment pipeline (link if interested, but I’ll explain: http://www.curefa.org/pipeline )

So the various drugs in development take different approaches to helping us. ANYTHING that could help is exciting, but some things are obviously…[Read more]

Hey Rich that is a great question. Obviously the mechanism of why Etravirine affects Frataxin regulation is unclear. So it makes sense to wonder if it could affect gene therapy. If you took them at the same time, it might. But you wouldn’t be taking them at the same time.

Etravirine isn’t a one-and-done drug. HIV patients have to con…[Read more]

That’s cool! Can anyone joining the WhatsApp group still post any updates here though? I like the forum for many reasons–including that the information people share is available for anyone to see 🙂

I’ve been going every Tuesday now since the start of the semester (mid-January). I joined the climbing club at my school. No one in the club has a disability but they were super excited that I wanted to give it a go.

Before starting I had no idea if I was going to be able to do it. I still walk but my peripheral sensation is almost completely…[Read more]

I can’t tell if I have FAtigue or if I’m just being overworked because of grad school. It’s probably both, lol.

I’ve never had coffee though… I don’t like the idea of relying on an external source of energy. My thoughts might change over time hahaha, but for now i just try to rest when I need it and stay as active as possible so I don’t have…[Read more]

Great article!!! I love both of ya 😀

Also it’s funny you chose the word steadfast. Was that intentional (referencing the Steadfast/Actimmune trials for FA)?

Omg, for the longest time I’ve thought “there must be a link here”! Like almost every FA’er I’ve ever talked to, I exhibit sleep behaviors suggestive of REM behavior disorder (walking, talking, moving etc…). I remember when I came across this paper I was even more convinced. I hope someone out there will do a study on FA sleep someday soon so we…[Read more]

Hi Chris! While the trials are ongoing I can’t speak to my individual experience as that could disrupt the clinical trial process, however I can say that the published results from the Part 1 trial showed 3 months of treatment with the active medication led to neurological improvements (measured by FARS) equal to reversal of 1 year of FA…[Read more]