Re: Jon Moulton: Have you heard any reaction to that article you posted?
I finally got around to checking to see which form Spinraza (Ionis's Spinal Muscular Atrophy drug) is:
"Nusinersen is a modified antisense oligonucleotide, where the 2’-hydroxy groups of the
ribofuranosyl rings are replaced with 2’-O-2-methoxyethyl groups and the phosphate linkages
are replaced with phosphorothioate linkages. Nusinersen binds to a specific sequence in the
intron downstream of exon 7 of the SMN2 transcript.
Phosphorothioate ...... the bad one. That's too bad. I read the side effects before I found this and they sure rang a bell! - Sounded like those of drisapersen. Too bad, but now I know why we are probably going to do our version at some point....even though Novartis now has Medell's one-shot SMA drug that looks like it works like a charm.