Treatment update again

I was hesitant to post here because i do not know the drug I will be taking as of yet. They have told me but I will have to ask again and I will one of these days. But anyway there os a doctor developing a drug specifically for SCA7. Currently I am in Maryland becaise a research center he works with has flown me out here to run tests and have me on file. When i do get this drug in 2018 they will document my progress. Eventually I’m sure this drug will be able to help all SCA. Again i will ask about the name I just know it will be injected into my eye to…and Im quoting…“help and maybe reverse some symptoms”. With any new drug things may not work out OR it just may very well be the treatment we’ve been looking for.

I know something about research and there are some questions you should be asking. First, is this study part of a clinical trial and, if so, what stage is the trial? Has safety of the drug been demonstrated? What are the risks? What is the probability of getting the drug vs. a placebo?
Can you read more about the study at clinical and, if so, how? How will you be monitored? What is the study sponsor’s responsibility to you in the event of adverse reaction?

These are questions off the top of my head. I also suggest you ensure, which you have probably done, that your neurologist is on board.

Although it may not sound like it, I am very pro-research and wanted, and failed, to get into a clinical trial myself. Just be sure of what you are doing.


Wow! I’m very squeamish about shots. I don’t have enough courage to go through an eye shot. I admire your bravery. I hope it’s safe & goes well.

What i know is the next step in the doctor getting the drug out there is human trials which me and 24 other people have been picked for. Im not sure about there being a placebo i havent heard anything about that. I will ask more about it but the good thing is it feels like im going in the right step. And it looks like as far as risks and side effects are concerned I will be a kind of a"guinea pig."

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It is nerve wrecking and i do feel a bit of pressure to get better…but thank you.

Great news, keep us updated!

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Please keep updating us. I would love to know :purple_heart:

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Hello everyone! Just got the good news that I will participating in a clinical trial using a drug called BHV-4157 (its main by-product riluzole). I start the frist week of April and will let everyone know of my progress. Hope do not get Placebo. Wish me luck…


Looks like you know more about your drug than I do. Good luck!

A good luck and keep us posted.
Very best wishes.Donna

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hey you @henry.n, how is the trial going?

Will start tomorrow!

Just passed 4 weeks and unfortunately nothing to write. There has been NO change in anything! Must have been in the Placebo group. Will update later when 3rd phase starts the beginning of April.

Hi Henry. My daughter is starting the same test in ucla starting next week. They did promise to give the real Meds after the 8 week trial is up. I believe it is to help slow down progression so I don’t know if you would feel different or if they test the rate of degeneration. I’m not sure yet. Did they tell you what you might feel? My daughter has sca8.

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Hi Cynthiafinnell,

Yep, I also will get the real stuff at the end of 8 weeks, (May 30). Just went in for my follow up and did a SAR test to see if there were any changes. The technician told me some of the results showed I went up and some I went down but I do not believe in the result. It is so subjective and I actually gotten worse. How can they say “my gait was a little better”!?!? They didn’t say anything on how I would feel. Will keep everyone posted.

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Hi Henry. I’m waiting for my daughter to get approved. The dr at ucla was feeling positive about this trial. It is at ucla. looking forward to hearing from you and your progress.

Hello, how is this trial going? :slight_smile: how are you guys feeling?

My daughter starts her trial on Friday. We are very excited and hopeful.

That’s awesome! I hope this will help your daughter, and eventually everyone who suffer from ataxia. Will you keep us posted about the results? :slight_smile:

Absolutely I will keep you posted.