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Dear all,

My 9 years old son has been taking Tamoxifen for 2 months now. He is taken care of by a wonderful team in Hadassah Hospital, Jerusalem.The drug considerably improved his condition: He can walk far longer, better and has more energy overall. And there are no side effects.

Just wanted to share.

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How is your son doing Sampreet.

Hope he is not on wheelchair.

So there won't be a complete cure for DMD?



Sampreet said:


Hi,I am the parent of a 16 year old with duchenne muscular dystrophy . I suggest you contact DART dystrophy annihilation research trust in bangalore if you are in India . If you need to know ,price of eteplirsen in Indian rupees is nearly 2 crore rupees per year. In us dollars it is 300000 dollars per year. In USA deflazacot cost is estimated to be around 89000 dollars. Ezutromid is considered only to help to reduce the severity of DMD it is not expected to cure the disease itself.
Sai Chand. Gurujala said:

Hi All,

EXONDYS 51 (eteplirsen) is approved by FDA in Sept'2016. I heard this medicine is not much effective for the treatment. Is this true? And does anybody know the price of it?

Now, Ezutromid and Tamoxifen are on clinical trials. As DMD is related to genetics and mutations, Does these therapies will cure all forms of DMD patients completely in future?

Deflazacort (Steroid), now approved in USA. In india, its price is very less. What is the cost for this steroid in USA?

Please, share.

No but smtc1100 is enough to prolong the time we need to find the best therapy to cure it.I think gene therapy is the most effective way to cure this disease, but the big size of the dystrophin makes it nearly impossible to deliver the required material to all the body. My son is not in a wheelchair but is finding it very difficult to walk.We use prednisone everyday.Please consult with your doctor about the best way to manage the conditions of your nephew.I think your nephew may benefit from steroids,but it is a very difficult decision to make and I suggest you consider the cons and benefits before starting him on steroids.I also want to tell you if you want to use steroids,both deflazacort and prednisone do not work same way as some kids see benefit in prednisone and none in deflazacort and vice versa.


Sai Chand. Gurujala said:

How is your son doing Sampreet.

Hope he is not on wheelchair.

So there won't be a complete cure for DMD?



Sampreet said:


Hi,I am the parent of a 16 year old with duchenne muscular dystrophy . I suggest you contact DART dystrophy annihilation research trust in bangalore if you are in India . If you need to know ,price of eteplirsen in Indian rupees is nearly 2 crore rupees per year. In us dollars it is 300000 dollars per year. In USA deflazacot cost is estimated to be around 89000 dollars. Ezutromid is considered only to help to reduce the severity of DMD it is not expected to cure the disease itself.
Sai Chand. Gurujala said:

Hi All,

EXONDYS 51 (eteplirsen) is approved by FDA in Sept'2016. I heard this medicine is not much effective for the treatment. Is this true? And does anybody know the price of it?

Now, Ezutromid and Tamoxifen are on clinical trials. As DMD is related to genetics and mutations, Does these therapies will cure all forms of DMD patients completely in future?

Deflazacort (Steroid), now approved in USA. In india, its price is very less. What is the cost for this steroid in USA?

Please, share.

Hi Sanja, sorry for the late answer. Actually yes, my son is still on deflazacort (every other day). I also give him idebenone (300mg daily). But in my son no existing drug has been better than tamoxifen so far.

Sanja Kogelnik said:

This is encouraging news. So this is yet another option for the treatment of DMD.

Now that your son is taking Tamoxifen does he take corticosteroids or specific vitamins such as enzyme Q 10, extract of green tea, fish oil ...?

Dear Galia,

thank you very much for sharing this news with us. You give me new strength.

Good luck ;)

Thanks Galia Ben!! this is very helpful information. 

Hello,

My son is suffering from Limb Girdle MD.This topic about Tamoxifen is really interesting because no treatments are underway for his condition.

Research was conducted on mdx mice so I don't understand  if this drug targets DMD muscles only or if it is for the pathological muscle in general. 

I realize that only a researcher could possibly answer this question so I would be really glad if Galia Ben or anyone else could provide some helpful info or help me get in touch with the researchers.

Thank you



Galia Ben Ishay said:

Hi Sanja, sorry for the late answer. Actually yes, my son is still on deflazacort (every other day). I also give him idebenone (300mg daily). But in my son no existing drug has been better than tamoxifen so far.

Sanja Kogelnik said:

This is encouraging news. So this is yet another option for the treatment of DMD.

Now that your son is taking Tamoxifen does he take corticosteroids or specific vitamins such as enzyme Q 10, extract of green tea, fish oil ...?

  • Dear Galia Ben Ishay, 3-year-old son was diagnosed with BMD.47-48 exons detected deletion.Doktor said that the BMD is light but I worry too much for my son .. I have read thrilledly about your writing on temoksifen and I will have questions, 1) how much ck has fallen since using tempexifen, what was the ck value before, now how much? 2) does your son continue to improve, what changes do you observe in his or her gait and movements? 3) Do you have information about the status of other patients using tempexifene? 4) cortisone medications have periodic improvements, are you the effect of these cortisone drugs or the effect of temoksifenin? Exactly how much ck has fallen, if you write the obvious changes you observed in your son ... I wish for urgent healing and patience .. Thank you in advance for your reply ..

Hi Antonis and Taylan,

Antonis: I cannot answer you answer but I think you may contact Prof. Olivier Dorchies from Switzerland who tried the Tamoxifen on DMD mice.

Taylan: My son's CPK has decreased a bit (about 300 less) yes he continues to improve slightly, I know that the other boys who've been taking it for longer than my son have improved or at least stabilized, some of them had their cpk levels reduced by half, as for the steroids, I decided to decrease the dose I give to my son to reduce side effects and I don't see any difference in terms of strength since I decreased them.

Thank you for your reply Galia,

Unfortunately Prof.Dorchies hasn't replied to my mail yet.

Could you provide some info as how to get in touch with some people from Hadassah medical centre?


Thanks galia for your reply .. We are glad you inform us about the developments frequently .. We fall in your level, we are waiting for the developments in your son curiously.

Hi Galia
In what dosage is he using tamoxifem... is it a prescription drug

Hi Vivek,

20mg a day in the evening. Yes it is a prescription drug.

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