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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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Good to hear about the Tamoxifen,

Is your son diagnosed with DMD.

Can we go for it.

Yes my son has DMD. And a very severe phenotype. Before starting the treatment he could hardly walk a few steps at home.

Thank you for your reply Galia.

Good to hear about your son improvement.

May i ask you, what was that phenotype and which exons. (Deletions or Duplications)

My nephew was diagnosed with DMD in 2016. He is 7.5. Duplication of exons 2 to 4.

Tamoxifen will cure DMD completely.

Please share

Dear Galia,

thanks for sharing this great news ;)

My son has deletion of exons 8 and 9. Anyway Tamoxifen is not mutation specific.

Thank for the great news, it seems to be part of Phase 1 of the this research:

https://www.clinicaltrials.gov

Let's all hope for the successful completions of all phases and with god grace all children are cured.

May be it completes in 2019

Michael Bazant said:

Thank for the great news, it seems to be part of Phase 1 of the this research:

https://www.clinicaltrials.gov

Hi Galia,

Thanks for sharing. Very exited to know that Tamoxifen is working. Are you giving it as part of the trial or on your own. What is the dose that you are giving. Did you observe any side effects. Did you see any reduction in fibrosis or any muscle development. Wondering if we can we start it on our own. I did not see any sites for the clinical trial. Is it going to be in Israel only or in the US also.

Regards

Rahul

Hi Rahul,

Actually he takes it through compassionate use in Hadassah hospital. He takes 20mg every evening. I can't see any side effect so far and none of the 3 children who've been taking if for about one year have experienced any side effect so far as well. I don't have much info from them but I know for sure  that the drug led to some improvement in their condition (reduction of cpk level and more energy overall for instance) and what is 100% sure is that none of them has deteriorated so far. The trial is currently ongoing and I still don't know when first formal results will arrive.

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 ...?

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,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.

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