I have CPT 2 deficiency, and I’ve been taking bezafibrate for almost 6 years as of today.
I understand that studies on bezafibrate are contradictory, but I decided to continue taking it because I think it is helping me.
Also, in-vitro, it was shown that when my fibroblasts were incubated with bezafibrate, fatty acid oxidation was (almost) normalized.
The daily dosage that was studied was 600mg a day. In my country, it used to be available in 200mg tablets, so I used to take 1 tablet 3 times a day.
Since about 3-4 years, it is now only available in SR 400mg tablets, so I usually take 1 tablet every 16 hours to reach a daily dosage of 600mg a day.
Let’s pretend that there was no doubt that bezafibrate 600mg a day was effective in CPT 2 deficiency. Does it make sense to take it every 16 hours, even though I’m not getting the same concentration of the drug reaching my blood throughout the day? Do you think I might still benefit from its ‘possible’ efficacy?
It is much simpler for me to take 1 tablet of bezafibrate SR 400mg every 24 hours. Do you think I might still get some benefit, if there is one, at a daily dosage of 400mg, or should I keep on taking 1 tablet every 16 hours?
Although potentially beneficial in short term, inducing mitochondrial biogenesis with bezafibrate worsened the metabolomic signature of mitochondrial disease, increasing FGF-21… since I’ve been taking for years, do you suggest measuring, monitoring metabolic biomarkers FGF-21, or others, to for possible toxicity? Which ones?
I’m hoping I could switch from bezafibrate to ren001 one day if there is some benefit, and if gets approved. I saw that there’s an ongoing phase 1 clinical trial. Is it possible to give a very rough estimation on how much time it would take for the drug to get approved, if nothing goes wrong?
Usually, how many years in general does it take for a drug to be approved, if it successfully passes all phases? Thank you!
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