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Is Translarna safe to take for g6pd deficiency?

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Posted on Wed, 11 May 2016
Question: HI my son has g6pd deficiency and has just been diagnosed with muscular dystrophy...type not confirmed yet.
Coq10 is taken for MD ..I was wondering if it is ok for him take it ? Do you know? Also there is a new drug called Translarna ... do you know if it would be ok to take? thanks XXXXXXX
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Answered by Dr. Olsi Taka (2 hours later)
Brief Answer:
Safe, benefit of translarna is questionable

Detailed Answer:
I read your question carefully and I understand your concern, I am reaaly sorry about your son's health issues.

Regarding the effect of CoQ10 on G6PD deficiency, you do not have to worry, that coenzyme does not play a role on it and can be safely taken.

As for Translarna....it is not listed among drugs who carry a risk for G6PD deficiency patients. However it is a new drug in the market and there is not a lot of experience with its use and perhaps the future may reveal new unknown side effects, however judging by the way it acts it shouldn't make any harm in G6PD deficiency.

As for its efficacy in MD though, it is questioned by many. It has been approved in Europe, but not in the US and not approved for reimboursement in Uk, because it is thought that studies up to now haven't really shown any substantial benefit, certainly not enough to justify its huge cost unfortunately.

I remain at your disposal for other questions.
Above answer was peer-reviewed by : Dr. Chakravarthy Mazumdar
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Follow up: Dr. Olsi Taka (31 hours later)
thank you for your prompt reply. it was a great help.
doctor
Answered by Dr. Olsi Taka (1 minute later)
Brief Answer:
You're welcome.

Detailed Answer:
I hope things work out for the best.
Above answer was peer-reviewed by : Dr. Chakravarthy Mazumdar
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Answered by
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Dr. Olsi Taka

Neurologist

Practicing since :2004

Answered : 3668 Questions

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Is Translarna safe to take for g6pd deficiency?

Brief Answer: Safe, benefit of translarna is questionable Detailed Answer: I read your question carefully and I understand your concern, I am reaaly sorry about your son's health issues. Regarding the effect of CoQ10 on G6PD deficiency, you do not have to worry, that coenzyme does not play a role on it and can be safely taken. As for Translarna....it is not listed among drugs who carry a risk for G6PD deficiency patients. However it is a new drug in the market and there is not a lot of experience with its use and perhaps the future may reveal new unknown side effects, however judging by the way it acts it shouldn't make any harm in G6PD deficiency. As for its efficacy in MD though, it is questioned by many. It has been approved in Europe, but not in the US and not approved for reimboursement in Uk, because it is thought that studies up to now haven't really shown any substantial benefit, certainly not enough to justify its huge cost unfortunately. I remain at your disposal for other questions.