Author: Jens-Ingo Farley
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What is BHV-4157?
2018-08-14. Trigriluzole was renamed to troriluzole. On 2018-11-02, most references to trigriluzole and BHV-4157 were removed from the Biohaven website. 2017-October: After SCA trials failed, Biohaven was quick to remove some SCA-related information from their website (hence, some broken URLs). See here. Later, they resumed testing after doubling the dose. 2017-April (or so): Biohaven removed…
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Stranger in a Strange Land
The world would be a strange place if everyone in it grokked the ataxia family of diseases. What does the average person have to gain by educating themselves on one rare disease, if it doesn’t affect them, and there are thousands of rare diseases? Nothing. I don’t know if this is a real quote, but…
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Muddled Thoughts on a Cure
I’ve never expected a cure for the SCA3 disease in my lifetime. Nevertheless, I’ve been thinking about it recently, especially in light of citalopram looking promising and some upcoming clinical trials. What are we talking about? Cerebellar degeneration. This is the fundamental issue with having SCA that causes external symptoms of gait ataxia and other…
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Unchained Malady
I was trying to think of funny alternative names for SCA, and this song title parody jumped out at me (being that DNA exists as chains of nucleotides), so I decided to write a complete song parody. Sung to the tune of Unchained Melody. Whoa, my alleles, my DNA I’m getting out of touch A…
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Citalopram and SCA3
Most technical papers on SCA are more advanced than I can comprehend. Every sentence can be chock-full of references that I don’t fully understand. This article takes an example of what looks like a promising SCA3 study involving drug repurposing and dissects it. Here’s the study, published 2015-09-15: https://www.researchgate.net/publication/282042707_Serotonergic_signalling_suppresses_ataxin_3_aggregation_and_neurotoxicity_in_animal_models_of_Machado-Joseph_disease Breaking down the summary Here’s a…
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Drug Repurposing
This is an outsider’s view of what drug repurposing is about. When a drug is approved by the FDA, it is approved for a specific purpose. When an FDA-approved drug is used safely for a purpose other than for what it was approved, that is referred to as using it off-label. If a drug is…
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Rarity Again (Neurologists)
As we know, though some deny it, the prevalence of spinocerebellar ataxia (SCA) is upwards of 5 per 100,000, or about 16,100 in the U.S. in 2015. Consider this: There are about 16,366 neurologists in the U.S.—about 5.1 per 100,000 (There are about 915,000 doctors in the U.S.—about 280 per 100,000) https://www.aan.com/PressRoom/home/PressRelease/1178 https://www.kff.org/other/state-indicator/total-active-physicians/ The prevalence…
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Prevalence and Incidence of Rare Diseases
I’ve now come close to doing something I thought was impossible in my first post, which is knowing where the prevalence of ataxia stands among a list of other rare diseases. I was drawn to this PDF because of the prevalence and incidence information it contains on 3,064 rare diseases, and I have made the…
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Hear Me Play the Piano
Playing the piano has been a lifelong learning experience. At least I was learning until the downturn due to having a rare disease called ataxia. More of my thoughts about having ataxia are all over this site and specifically here on the topic of playing the piano. You can also click on About Me or…
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Playing the Piano
I started playing the piano at about age nine. At ages eleven and twelve, I won two small, local, age-graded piano competitions. Though I had some talent, I was by no means gifted, and I spent my adult life continuing to play the piano and face my limitations. I have played classical music, from 18th-century…