there is something much more important out there today
I am thinking of 4 words
I love word games
and now for the asteroid ...
"2 Phase Ib and 2 Phase IIa clinical trials (MDEX Consortium in collaboration with Sarepta Therapeutics; a Dutch Consortium) demonstrated that delivery of antisense oligonucleotides (AOs) to mediate exon skipping of exon 51 were able to return specific DMD mutations in-frame (~13% of all mutations) leading to new dystrophin protein expression after intramuscular and systemic delivery. The Dutch study of repeated 2-O-methylated phosphorothioate (2OMe) AO administration suggested limited efficacy after 5 weeks of treatment. Our MDEX Consortium study using a morpholino (PMO) AO demonstrated a clear dose response, robust dystrophin restoration and reduction of muscle inflammation after 12 weeks at doses up to 20mg/Kg, with no drug related adverse events. This, and preclinical studies focused on level of protein expression, clearly indicate that PMO have a superior therapeutic index compared to 2OMe."
now what, exactly, precisely, is this 2OMe thingie?
I am thinking of 2 more words
this is very serious
if you dare to go outside, wear a criptonite hard hat, cuz we are waaaaaaaaaay past tea leaves now
And the hits keep coming. Great news. This drug is a clear winner. I especially love the "no drug related adverse events" part. This drug is so superior in every way to Proensa. I haven't heard ANY feedback form boys in the Proensa trials. Menawhile the parent of the boys in the Eteplirsen trials just cannot keep quiet about the amazing progress of their son's.
"The progression of clinical trials is shown for the two key antisense oligonucleotide chemistries: 2′-O-methyl phosphorothioate (2OMe) antisense oligonucleotides, which were developed initially by Prosensa (now in partnership with GlaxoSmithKline (GSK)); and phosphorodiamidate morpholino (PMO) antisense oligonucleotides, which are being developed by the MDEX Consortium and AVI BioPharma."
holy crap I just got hit in the buttocks by an asteroid that bounced off johnnyk's head