Immigrant scientist studying amyloidosis. Not a physician. He/him

USA
Joined October 2009
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): mdpi.com/2673-6357/2/4/42
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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?
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Gareth Morgan retweeted
DepleTTR-CM trial is now on clinicaltrials.gov Phase III trial of ALXN2220 (NI006) vs placebo Fibril remover ( phase I results nejm.org/doi/full/10.1056/NE… ) 1000 patients, ATTR-CM, loop diuretics, NTproBNP > 2000. PEP: ACM+CV events classic.clinicaltrials.gov/c… #CardioTwitter
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15 cases of AGelsolin amyloidosis: largest series in the US. 20% renal involvement, 7% cardiac involvement besides cranial, peripheral and autonomic neuropathy. @lis589 @vsanchorawala pubmed.ncbi.nlm.nih.gov/3714…
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With apologies to Yiying Lu.
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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 scripps.edu/news-and-events/…
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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. investor.bridgebio.com/news-…
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Kudos to @BridgeBioPharma for sticking with the trial after the "disappointing" (but not really surprising) 12-month data
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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. investor.bridgebio.com/news-…
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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. amyloidosisforum.org/attr-dr…
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A review of our work to develop stabilizer drugs for AL amyloidosis is out today in Current Opinion in Chemical Biology: authors.elsevier.com/c/1hCX7… Highlights the work of @ScrippsGradPrgm alum Nick Yan, who developed high-efficacy molecules against a non-traditional drug target
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ARC is pushing research forward today, on #NationalAmyloidosisDay, and everyday. Our programs like the #AmyloidosisForum are a model for collaboration garnering support from the @NIH and Members of Congress like @JakeAuch: arci.org/nih-grant-2023/
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Finally published a real "research parasite" paper (remember those?) reanalyzing data from @theMMRF CoMMpass study to identify complete light chain variable domains: frontiersin.org/articles/10.…
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These sequences will help us to understand why some antibody light chains can form amyloid whereas most don't. The sequences are deposited in our AL-Base resource: wwwapp.bumc.bu.edu/BEDAC_ALB…
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Great work from lead author Allison Nau and my colleagues at BU. We're grateful to @theMMRF for access to the data, and to the patients who volunteered for the original study and made it possible.
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I'm quoted in this great article about systemic amyloidosis by @amberldance which explains how our research @BU_Amyloidosis fits into the bigger medical challenge. Also featuring @i_lousada of @Amyloidosis_ARC knowablemagazine.org/article…
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NEW: Amyloid diseases that affect the brain, such as Alzheimer’s and Parkinson’s diseases, receive the lion’s share of attention — but there is growing medical appreciation that amyloid diseases affect tissues beyond the brain. ✍️ @amberldance knowablemagazine.org/article…
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Gareth Morgan retweeted
Proud of such an excellent team effort- will done snd thanks to all!
It's lovely to finally see this beautiful work from @milkowilko @R_U_Gallardo and friends on β2m amyloid out today in @NatureComms #AmyloidLego @AstburyCentre @Astbury_BSL @RadfordLab @wellcometrust @The_MRC #OpenAccess at rdcu.be/c6JU7
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