With Professors Jeff Kelly and Joel Buxbaum at Scripps Research, I wrote about our goal of creating drugs to stabilize antibody light chains and suppress their misfolding and aggregation, as a potential therapy for AL amyloidosis (open access): https://t.co/aqgID5cx0u
Fantastic to see our competitive, market-driven healthcare system in action as BridgeBio launches a newly-approved TTR stabilizer molecule at a list price that's [checks notes] 10% higher than the indistinguishable competitor?
DepleTTR-CM trial is now on https://t.co/5QFuhycc8b
Phase III trial of ALXN2220 (NI006) vs placebo
Fibril remover ( phase I results https://t.co/2D6HAYg5Np )
1000 patients, ATTR-CM, loop diuretics, NTproBNP > 2000.
PEP: ACM+CV events
https://t.co/RaxczA7rQ7
#CardioTwitter
Mechanisms on pathophysiology of amyloid fibrillogenesis form @BU_Amyloidosis@wittyremarkhere
Kinetic evidence for multiple aggregation pathways in antibody light chain variable domains https://t.co/Ym7sHKndNm
@MarkMcConway1@awechalekar Twitter is bad at context and detail. The paper says that there's not enough evidence to support the claim (by other authors in a recent study) that bortezomib is nephrotoxic, and that substantial earlier evidence points to it being safe. https://t.co/oxfTHbn1MT
15 cases of AGelsolin amyloidosis: largest series in the US. 20% renal involvement, 7% cardiac involvement besides cranial, peripheral and autonomic neuropathy. @lis589 @vsanchorawala
https://t.co/rsdBiO37et
The Scripps Research community mourns the passing of Michael Oldstone, MD, esteemed virologist and professor emeritus, who helped establish the viral pathogenesis field and shaped our understanding of host immune responses https://t.co/v21IUqSVii
Good to see that acoramidis met its endpoints for ATTR amyloidosis in the ATTribute-CM trial. Important that there's a significant benefit even in a cohort that was healthier than in previous ATTR trials.
https://t.co/JK0M5VHbSY
Online now (@ISA_Amyloidosis)! S/o to @sabrina_ghosh for her hard work during the pandemic (this was her UNDERGRAD❗️thesis)! HUGE thanks to the 🐐 mentor @DrGJMurphy for letting me play junior PI (my first (and hopefully not last) senior author paper🥸)
https://t.co/ljBie1qyMO
Big day for patients with ATTR amyloidosis, as the topline results of ATTRIBUTE-CM are positive!
Acoramidis showed significant benefit for the hierarchical composite of all-cause mortality, CV hospitalization, NTproBNP, and 6MW distance.
https://t.co/IKU5iJ5s3G
@vineetcardio@VincentRK Normal FLC + positive scintigraphy: likely ATTR
Abnormal FLC + negative scintigraphy: likely AL
Abnormal FLC + positive scintigraphy: could be either, MUST verify with typing
Normal FLC + negative scintigraphy: not amyloid, or a rarer variant like AApoA1
https://t.co/TVbpyGNH0G
The first generation of drug approvals in #ATTR#amyloidosis has reshaped the research landscape. This month's #AmyloidosisForum will consider how to effectively design trials against a backdrop of treatments that slow progression of the disease.
https://t.co/R7suiCWCGW
A review of our work to develop stabilizer drugs for AL amyloidosis is out today in Current Opinion in Chemical Biology: https://t.co/RRgdv6Tcn6
Highlights the work of @ScrippsGradPrgm alum Nick Yan, who developed high-efficacy molecules against a non-traditional drug target
Congratulations to Dr Elena Klimtchuk of @BU_Amyloidosis in elucidating mechanism of amyloid fibrillogenesis.
Role of complementarity-determining regions 1 and 3 in pathologic amyloid formation by human immunoglobulin κ1 ligh… https://t.co/bVpw7Y5sj6