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Posted

Hello all,

One of my good friends son has been diagnosed with 'Duchenne muscular dystrophy' after researching the condition for a few days I see no real cure for this unfortunately however there is a drug 'Deflazacort' a type of steroid which can seriously prolong the condition and help maintain muscle deterioration.

He is finding this drug very hard to get in the UK even from a doctor, I was wondering, is it possible to buy this in Thailand OTC? I know its a long shot but I told him I would find out,

Cheers

Posted

If this is for use in the UK, then I am extremely surprised that he says he's having difficulty in obtaining it.

You should advise your friend to contact the Neurologist who confirmed the diagnosis, & ask him for his assistance.

Posted

Steroid drugs cannot be bought OTC in Thailand, they require a prescription. These are very serious medications with serious side effects and it needs a skilled physician to determine whether in a specific case the potential benefits outweigh the risks. In addition, dosage has to be highly individualized. Only a neurologist with experience managing muscular dystrophy would be able to do this properly.

Deflazacort is in the same drug family as prednisolone which equally effective and less expensive, and probably what your friend's son has been given. Some studies suggest a better side effect profile for deflazacort (especially regarding obesity, a common side effect of prednisoilone) but this is not yet certain, and some skepticism should always be maintained about claims for "new" versions of older drugs that are long off patent. See this 2012 study comparing the two drugs: http://commons.pacificu.edu/cgi/viewcontent.cgi?article=1308&context=pa

Deflazacort is not yet approved in the US (but FDA approval in progress). I am not sure if it is approved yet for use in the UK -- as there is a current clinical trial ongoing for it, maybe not -- but if it is, it is likely restricted by the NHS to cases where there is a specific reason to switch from prednisolone, as it is very expensive while prednisolone is available in many generic forms.

There is far more experience with the use of prednisolone, which IMO is in itself is a good reason to treat with it, at least initially. Among other things, the optimal dosage schedule is still being determined for the newer drug. Nothing suggests that deflazacort will significantly improve outcomes over prednisolone, though in some cases it may be better in terms of specific side effects.

Unless your friend's son is suffering intolerable side effects from treatment with conventional steroids, there is no reason to try to push for treatment with deflazacort.

Posted

Steroid drugs cannot be bought OTC in Thailand, they require a prescription. These are very serious medications with serious side effects and it needs a skilled physician to determine whether in a specific case the potential benefits outweigh the risks. In addition, dosage has to be highly individualized. Only a neurologist with experience managing muscular dystrophy would be able to do this properly.

Deflazacort is in the same drug family as prednisolone which equally effective and less expensive, and probably what your friend's son has been given. Some studies suggest a better side effect profile for deflazacort (especially regarding obesity, a common side effect of prednisoilone) but this is not yet certain, and some skepticism should always be maintained about claims for "new" versions of older drugs that are long off patent. See this 2012 study comparing the two drugs: http://commons.pacif...1308&context=pa

Deflazacort is not yet approved in the US (but FDA approval in progress). I am not sure if it is approved yet for use in the UK -- as there is a current clinical trial ongoing for it, maybe not -- but if it is, it is likely restricted by the NHS to cases where there is a specific reason to switch from prednisolone, as it is very expensive while prednisolone is available in many generic forms.

There is far more experience with the use of prednisolone, which IMO is in itself is a good reason to treat with it, at least initially. Among other things, the optimal dosage schedule is still being determined for the newer drug. Nothing suggests that deflazacort will significantly improve outcomes over prednisolone, though in some cases it may be better in terms of specific side effects.

Unless your friend's son is suffering intolerable side effects from treatment with conventional steroids, there is no reason to try to push for treatment with deflazacort.

Thank you Sheryl, useful information as usual and much appreciated, I have relayed this information onto him, thanks alot.

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