Hello all, does anybody have experience in taking this drug? There is no mention on this site. I have been now diagnosed with NPC-1 and am going on a clinical trial for Arimoclomol. Will let everybody here know the outcome.

Never heard of it. Does it work for SCA? Let us know if the drug has any effects.

:thinking:I’ve never heard of this before…I did find this…I recall there being a mention of a specific drug re NPC-1 but it wasn’t this one.

But now, the FDA has officially rejected the Danish biotech’s Niemann-Pick disease type C (NPC) drug candidate arimoclomol, sending the shares crashing back to Earth.18 Jun 2021 › fda…

That is very disturbing! The EAP sponseor is Orphazyme. I hope they are not Fierce Biotech, will research. Thanks again

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This might be helpful !

Dr Oswal explains that “the body salts bring about the necessary changes in neuro- transmission while the herbal extracts act as a catalyst”, he believes that G-therapy can improve speech, concentration, communication, sleep, motor co-ordination, it can reduce spasticity, involuntary movements, hyperactivity and tantrums.
Dr Oswal doesn’t claim to have found a miracle cure; he suggests that G-therapy is more efficient when used in conjunction with other therapies.

G-therapy is a controversial treatment: since the beginning of the nineties numerous articles in the press have reported cases of children whose condition improved greatly thanks to the therapy; Dr Oswal claims that none of the 1700 children he treated with the G-therapy showed any signs of side effects, he also estimated a 70% rate of improvement in “reduced hyperactivity”, “improved speech” and “improved motor control”, and that the results were visible within twelve weeks. According to the Cerebra (previously known as The Rescue Foundation, a charity which focuses on brain injuries), more and more parents in the UK are interested in this treatment. However after several years no one is able to explain exactly how the therapy works, there is a lack of evidence: there has been no clinical trial so far to prove the benefits and safety of the therapy.

Practical facts: The duration of the therapy varies according to the condition of the child. It can take up to 12 weeks before the therapy starts working. The cost of a 6 months supply is £700.

Do let us know how it works out.

Hello everybody! Just had my 1 month review on this drug specifically for NPC-1 and have nothing to report. At least I am not experiencing any side effects. We shall see in 3 months!

:slightly_smiling_face: Time will tell, and as you say ‘ no sideffects’ is good news…

Just updating everyone on my use for Arimoclomol. I completed 247 days and stopped. I am currently gong to try Hydroxypropyl-beta-cyclodextrin for NPC1, will keep update how it goes.

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:thinking: How will this help NPC-1
Is is to aid absorbance of other meds

:slightly_smiling_face: I found this…it explains the link…

Studies showed that 2-hydroxypropyl-β-cyclodextrin (HPβCD) alleviates the cholesterol accumulation defect in animal model and has been approved for a phase 2b/3 clinical trial for NPC. HPβCD is known to bind cholesterol; however, the mechanisms how HPβCD mediates the exit of cholesterol from the LE/LY compartments are still unknown. Further, another cyclodextrin (CD) derivative, 2-hydroxypropyl-γ-cyclodextrin (HPγCD), was shown to reduce intracellular cholesterol accumulation in NPC patient cells and NPC mice.

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I had my first infusion and didn’t go well. I woke up the following day with bad hearing loss. I’m wondering should I continue with study? I’m doing it for the kids. It is a bad disease and if I can help in any way… They say I “should” get my hearing back but not convinced. It was tough driving back home with my wife not to mention everything on the TV MUST be in closed captions.

:confused: That’s very disappointing Henry…personally I think you should be advised by your Neurologist
I think you were brave to contemplate a study…no-one knows exactly what ‘side effects’ could occur and whether they’ll be permanent…it’s a case of fingers crossed

:slightly_smiling_face: Hopefully hearing will return to normal very soon…it could simply be due to your body adjusting to the infusion

(I had a rare bad ‘occurrence’ myself with my first session of chemotherapy…and it took some persuading to have me continue…so I empathise)

Thanks Beryl for the kind words. The Neurologist at the clinic couldn’t be more thorough and helpful. I am scheduled for (2) MRI’s for my brain and my ears. My thoughts are exactly as your “my body adjusting to the infusion”. I want to know, did you continue with chemotherapy and did it help?

:slightly_smiling_face: Yes …I completed the course, and it did help