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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. 

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