Ataluren 2b Trial Patients outside US and Canada

Hi,

 

My son took part in the 2b trial in UK. We are aware that ptc is looking at the possibility of the drug access for boys who took part in this trial.I am informed by ptc that they are only looking at access to Ataluren for patients in US and Canada and the responsibility to do the same for patients elsewhere lies with Genzyme.

 

Our details have been passed on to Genzyme and now the decision is upto them not ptc considering we only went in trial in the hope of definately getting the drug in the extension trial.Ptc also very rapidly recruited our boys for the trial internationally.

 

Please email both ptc and genzyme if your son took part in this trial in other countries than US and Canada as we do not want our sons treated differently .All patients deserve to be treated the same.

 

Please email Genzyme at eumedinfo@genzyme.com and dgoetz@ptcbio.com to voice for your son.

 

Please also email me your contact details at riasunita@yahoo.co.uk so we can put together one voice and a plan to move together if need be.

 

Please do not delay.We do not want our son left behind.

 

Regards

 

Neeraj  

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I have just learned about my son and am still in the process of understanding what can be done for him. What is Ataleuren? Is Ataleuren and AVI 4658 the same? To what extent is it helpful? How can it be made available? what will be the maximum time in which it will be commercially available to all?
Hi Pooja,

First I hope you and your family is well.Ataluren is a potential trearment in the advanced stages for dmd bmd caused due to nonsence mutation or also called stop codon or premature stop signal.Find out how is yous sons dbmd caused also check ptcbio.com site.

Regards

Neeraj
hi

i was on the trial. yes everybody has to join . all the kids on the trial deserve to have the same following treatment. An other solution would be unbearable

best

françoise

neeraj said:
Hi Pooja,

First I hope you and your family is well.Ataluren is a potential trearment in the advanced stages for dmd bmd caused due to nonsence mutation or also called stop codon or premature stop signal.Find out how is yous sons dbmd caused also check ptcbio.com site.

Regards

Neeraj

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