Replying to @Lpa_Doc
Thanks Sam, interesting point about the LDL-C baseline being quite low. Still, wouldn't the FOURIER (O'Donoghue et al) data indicate oppty for MACE reduction even when LDL-C is well-controlled?
ahajournals.org/doi/10.1161/…
Replying to @ethanjweiss
Re: #2, the O'Donoghue analysis of the FOURIER trial shows residual benefit from lower Lp(a) even when LDL is well-controlled. As Sam pointed out, the LDL-C in HORIZON may have been even lower, but that is a matter of degree, not that there is no benefit from Lp(a) lowering.
ahajournals.org/doi/10.1161/…
$amlx great comeback story.
I hope they come back to ALS at some point!
Remarkable data for kids with SCD and B-thal.
nejm.org/doi/full/10.1056/NE…
jamanetwork.com/journals/jam…
Dropoff in overall Rx is not surprising given it's curative but this has still been a massive class of drugs over the past ~12 years and the public health impact isn't discussed enough.
Worth remembering this molecule is a macrocyclic peptide with a 37-member ring. Impressive work by Merck’s process chemists.
$MRK oral PCSK9
businesswire.com/news/home/2…
Nice review article on Lp(a)
jamanetwork.com/journals/jam…
Amazingly, much of this (excellent) empirical research was PREDICTED by a mathematical model of Huntington Dz onset from way back in 2007!!
The observation that HTT homozygotes have similar age of onset as hets led to concept of a CAG length threshold.
pmc.ncbi.nlm.nih.gov/article…
New findings reveal surprising new details about how an inherited genetic mutation causes Huntington’s disease, transforming understanding of the disorder. broad.io/HDNews
Replying to @Jn66039675 @JacobPlieth
They'll get some responses but what's their TI? Released clinical data on MRTX molecule is still very limited. This is a dirty MoA.
(From MRTX paper last year)
Replying to @Prof_Oak_
Good observations about the importance of B cell depletion in tissues and naive B cell repopulation.
I think the data on TCE B cell depletion is less clear though. Blincyto B cell depletion in blood from SSc pts is transient.
And CLN blood B cell depletion in NHL looks variable
We had a moving ceremony with Tanzanian sickle cell kids earlier today as @sickleforward donated testing kits to local health authorities
Gear check underway for Kilimanjaro climb starting tomorrow. @ldtimmerman @SickleForward
givebutter.com/TimmermanTrav…
Less than one month away from the Kilimanjaro climb for Sickle Forward and I’m in the home stretch to raise funds to meet my $50k goal.
Help me get over the top! Donate to Sickle Forward here: givebutter.com/TimmermanTrav…





















