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Okay I am confused... I thought my son Trey was a canidate for this drug since he needs the exon 51 skip...so I thought that he was one of the 13% that this drug could possibly help...
BUT I just talked to Debra Miller at CureDuchenne (and I could tell she was extremely busy) but she said that the Ataluren was for Stop Codons, not deletions...????
Were we accidently talking about 2 different things at one time, or am I thinking correctly that this drug could help Trey who has a deletion of exon 52?
So confused...Cori
Yes, two different possible treatments for subsets of patients:
Ataluren is a drug for stop codons (~13% of DMD patients). Trials are going on in the US as well as other countries around the World.
Exon skipping is what Trey needs. Skipping exon 51 might become therapeutic for ~13% DMD patients. This, if approved, will be IV or subcutaneous injection (not pill). The exon skipping trials do not have FDA approval to start in the US at the moment.
Those % are approximate.
Cori said:Okay I am confused... I thought my son Trey was a canidate for this drug since he needs the exon 51 skip...so I thought that he was one of the 13% that this drug could possibly help...
BUT I just talked to Debra Miller at CureDuchenne (and I could tell she was extremely busy) but she said that the Ataluren was for Stop Codons, not deletions...????
Were we accidently talking about 2 different things at one time, or am I thinking correctly that this drug could help Trey who has a deletion of exon 52?
So confused...Cori
Ofelia,
Okay our genious =), so Trey is NOT a canidate for the Ataluren with having a 52 deletion? So approx 13% of the boys have stop codons and 13% of the boys need exon 51 skipped? Those are 2 completely different things, am I understanding that correctly? The stop codons have a 'wrong letter' need Ataluren, whereas the boys that need exon skipping (need prosensa/AVI) are missing a letter? Sorry I guess I have been following the researching thinking that Trey was a canidate for this drug (forget the flipping price tag, that is ridiculous! grrr) and I guess I possibly have been SO wrong. sigh.
Ofelia Marin said:Yes, two different possible treatments for subsets of patients:
Ataluren is a drug for stop codons (~13% of DMD patients). Trials are going on in the US as well as other countries around the World.
Exon skipping is what Trey needs. Skipping exon 51 might become therapeutic for ~13% DMD patients. This, if approved, will be IV or subcutaneous injection (not pill). The exon skipping trials do not have FDA approval to start in the US at the moment.
Those % are approximate.
Cori said:Okay I am confused... I thought my son Trey was a canidate for this drug since he needs the exon 51 skip...so I thought that he was one of the 13% that this drug could possibly help...
BUT I just talked to Debra Miller at CureDuchenne (and I could tell she was extremely busy) but she said that the Ataluren was for Stop Codons, not deletions...????
Were we accidently talking about 2 different things at one time, or am I thinking correctly that this drug could help Trey who has a deletion of exon 52?
So confused...Cori
You got it. Our sons have a deletion of one exon or more and are NOT candidates for Ataluren. Some boys with stop codon can benefit from exon skipping though, so the subsets are not completely distinct. Exon skipping 51 is what might work for Trey and Robert -- trials are only in Europe at the moment.
Cori said:Ofelia,
Okay our genious =), so Trey is NOT a canidate for the Ataluren with having a 52 deletion? So approx 13% of the boys have stop codons and 13% of the boys need exon 51 skipped? Those are 2 completely different things, am I understanding that correctly? The stop codons have a 'wrong letter' need Ataluren, whereas the boys that need exon skipping (need prosensa/AVI) are missing a letter? Sorry I guess I have been following the researching thinking that Trey was a canidate for this drug (forget the flipping price tag, that is ridiculous! grrr) and I guess I possibly have been SO wrong. sigh.
Ofelia Marin said:Yes, two different possible treatments for subsets of patients:
Ataluren is a drug for stop codons (~13% of DMD patients). Trials are going on in the US as well as other countries around the World.
Exon skipping is what Trey needs. Skipping exon 51 might become therapeutic for ~13% DMD patients. This, if approved, will be IV or subcutaneous injection (not pill). The exon skipping trials do not have FDA approval to start in the US at the moment.
Those % are approximate.
Cori said:Okay I am confused... I thought my son Trey was a canidate for this drug since he needs the exon 51 skip...so I thought that he was one of the 13% that this drug could possibly help...
BUT I just talked to Debra Miller at CureDuchenne (and I could tell she was extremely busy) but she said that the Ataluren was for Stop Codons, not deletions...????
Were we accidently talking about 2 different things at one time, or am I thinking correctly that this drug could help Trey who has a deletion of exon 52?
So confused...Cori
We are definitely anxiously awaiting the results of the trial and of course the FDA approval. I had no idea it would cost that much, though. I don't see how it could be morally justifiable in any way. I mean...that's $410 a day! Our neurologist said that the drug is for sale somehow already at $280 a pill. Of course not covered by insurance since it's not FDA approved yet. I did find it online at that price, too.
I am totally confused but excited also. Would this help my grandson?, he has deletions 46-55.
Tracey Hartz said:We are definitely anxiously awaiting the results of the trial and of course the FDA approval. I had no idea it would cost that much, though. I don't see how it could be morally justifiable in any way. I mean...that's $410 a day! Our neurologist said that the drug is for sale somehow already at $280 a pill. Of course not covered by insurance since it's not FDA approved yet. I did find it online at that price, too.
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