Ataluren at low dose - Obviously a success!
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Bernardo A. Iriberri
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Ataluren at low dose - Obviously a success!
Started this discussion. Last reply by Linda Owens Jun 5, 2010. 24 Replies 0 Favorites
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Ataluren Trials - After the Conference Call
Started this discussion. Last reply by Bernardo A. Iriberri Mar 11, 2010. 7 Replies 0 Favorites
I have read the notes on Friday's call with PTC, which were posted yesterday on PPMD's main website, especially Sharon Hesterlee's useful comments. I've also read Pat Furlong's blog and her words…Continue
Tags: Ataluren
Bernardo A. Iriberri's Page
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Bernardo A. Iriberri replied to Blazena Kaderabkova's discussion 'Arbekacin as a therapeutic readthrough inducer for treatment of nonsense mutation-mediated Duchenne muscular dystrophy'
Well, I have to reply to my own message, because I've just checked Arbekacin out and it turns out to be just another ultra-potent antibiotic, with the same toxicity issues that made gentamycin useless for this therapy.
This is from the…
Bernardo A. Iriberri replied to Blazena Kaderabkova's discussion 'Arbekacin as a therapeutic readthrough inducer for treatment of nonsense mutation-mediated Duchenne muscular dystrophy'
Thank you Blazena. I had never heard about negamycin or Arbekacin. I knew of course about gentamycin, but ruled it out in my mind because of the well reported adverse effects. My son has a nonsense mutation and we have been waiting for nearly nine…
Bernardo A. Iriberri replied to Trinh Nguyen's discussion 'Stop Codon Mutation'
Hello Blazena,
Yes, I've read the announcement that Genzyme is withdrawing from a deal to sell Ataluren around the world (outside the US), but they are retaining an option to get back into the deal at a later time. In other words,…
Bernardo A. Iriberri replied to Trinh Nguyen's discussion 'Stop Codon Mutation'
Hello Andrea,
Ataluren is designed to overcome any stop codon, regardless of the exon. My son has a stop codon mutation in exon 7, so I've been reading all about PTC124 (Ataluren) since I've heard about it 8 years ago. Unfortunately…
Bernardo A. Iriberri replied to Trinh Nguyen's discussion 'Stop Codon Mutation'
Thanks again! Let's see what comes out of the combined efforts of academic researchers and the pharma industry.
Bernardo A. Iriberri replied to Trinh Nguyen's discussion 'Stop Codon Mutation'
Hello Jon,
First of all thank you so much for explaining exon skipping so well, and thanks a lot also for the link to that chart.
My son has a nonsense, stop codon mutation in exon 7, so I understand he would need to skip exons 6, 7 and…
Bernardo A. Iriberri replied to Luis MErino's discussion 'Athena test came back negative, biopsy confirmed DMD'
Hi Luis,
My son's DNA was tested first through the more conventional ways (PCR) and no duplication or deletion was found, Then we started reading about the so called "DOVAM" tests (Detection of Virtually All Mutations). We live…
Bernardo A. Iriberri commented on Pat Furlong's blog post 'PTC Therapeutics—Day of Dialogue'
Thank you Sharon. It's clear now. I hope they get the approval soon.
Bernardo A. Iriberri commented on Pat Furlong's blog post 'PTC Therapeutics—Day of Dialogue'
Hi Sharon. I didn't understand Ofelia's question quite well, and I also remember myself that when the earliest reports came out, it was already noted that there seemed to be more distrophin in the cells of those receiving the low dose…
Bernardo A. Iriberri favorited Pat Furlong's blog post 'PTC Therapeutics—Day of Dialogue'
Ataluren Trials - After the Conference Call
I have read the notes on Friday's call with PTC, which were posted yesterday on PPMD's main website, especially Sharon Hesterlee's useful comments. I've also read Pat Furlong's blog and her words about this community's own "hurricane" are indeed moving to all of us. I thought it might be interesting to share with you the outcome of a quick search of Ataluren's clinical trials in the Government website www.clinicaltrials.gov, and the status of these…See More
Discussion posted by Bernardo A. Iriberri
Profile Information
- About me:
- I am a 55 year old attorney from Argentina, father of a boy with DMD
- About my family:
- My former wife and I have three children, two daughters aged 24 and 21 and a son who is 19 years old
- Name(s) of child(ren)/individual(s) with Duchenne:
- Hernán Iriberri
- Age(s) of child(ren)/individual(s) with Duchenne:
- 19 or older
- City and State:
- Buenos Aires
- Country:
- Argentina
Comment Wall (1 comment)
- At 9:31am on March 10, 2010,
Holly Now said… - Did or is your son taking steroids?
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