Arimoclomol

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jethro

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PALS
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09/2017
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HR
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ZAGREB
i dont believe in any drug that can act directly on a cause, but this one is, at least, closest to cause.
it can provide cleaning of misfolded proteins, hence enable autophagy which is impaired.
any info about trial?
trial is over.
 
It finished just under 2 months ago. No results yet. Hopefully soon
 
Is this treatment aimed at the SOD1 mutation or all ALS patients?
 
The original trial was sod1. The one that just finished was all. The SOD1 trial had some positive results
 
first mice (22% prolonged life+mobility), then humans. waiting for results in humans. it is very interesting that such a wide trial (29 locations - 9 usa, 2 canada, rest is EU) just pass through background door, 231 participants. i read info from a pals in trial (2, one was placebo, obviously) that it reduced his fasciculations a lot (if i understood well)
 
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ough... i forgot something extremely important: 1. trial with a humans (38 participians) - dosage was 1 x 200mg x 3/day, this time is/was 400mg x 3 /day. 2 x higher dosage!!!
 
i talk to lady on a trial in EU. she was eligible @44 alsfrs-r when she started. after 23 months on arimoclomol, her alsfrs-r is 35.
she continued to use a drug after trial.
not enough? ok, ill investigate further.
is there anyone who was on trial to share experience?
 
thanks laurie,
your comments are always precious. cytrx sold it to Orphazyme ApS 2 years after that article. with 3 x 400 g trials are not performed before. just study with mice, 3 x 200 mg for sod1 pals, II phase, recently. frankly, i doubt "same dose fits all" . it is oral drug, should be cheap (at least, generic drug should be affordable), does no harm... what i read, it is closest to als cause. read 3 comments (by now). 1. no changes (probably placebo), 2. rush, sickness, less fasciculations 3. significantly less fasciculation and progression.
time will tell
 
Yes, that's right, it was sold and development has been slow as frequently happens with cash-strapped small mfrs. But it was wise given the lack of toxicity seen in previous trials (three indications besides ALS in development), to amp up the dose, since response has been dose-dependent in earlier trials.

Not sure what you mean by generic drug. It will be branded at launch and go generic once exclusivity period expires.
 
indians and chinese pharmacy can make replica of any drug giving it other name with exactly same composition. it is not philosophy anymore. same was with edaravone. it will gain it's CAS number as soon as fda approves it. just like viagra, via-agra, kamagra...
 
Has anyone found anyone in this trial that can report a benefit? I've only found 1 person that reported a benefit. He claimed his twitches reduced and believes it slowed his progression. Any more than that? It seems we should be able to locate a few more given the number of patients and trial locations.
 
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