Podcasts about attr

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into a series of events that underscore the dynamic nature of these industries, characterized by scientific advancements, strategic partnerships, and regulatory milestones. Johnson & Johnson has made headlines with its substantial $785 million upfront payment to Sail BioMed for an in vivo CAR-T cell therapy deal. This agreement includes an option for Johnson & Johnson to acquire Sail BioMed for $2.58 billion, underscoring the sustained interest in cell therapies, particularly for autoimmune diseases. In vivo CAR-T therapies represent a significant leap forward by modifying T cells directly within the patient's body. This method offers potential advantages over traditional ex vivo techniques by simplifying the manufacturing process and potentially reducing both costs and time to treatment. Such transactions highlight Johnson & Johnson's commitment to expanding its cell therapy portfolio, which could significantly enhance patient care by making advanced treatments more accessible. Eli Lilly and Resilience have announced a significant $750 million investment aimed at boosting the production of diabetes and obesity medications in the United States. This move comes as a response to the global rise in these conditions, necessitating increased production capacities to meet growing demand. The focus on injectable drug devices emphasizes efforts to improve drug delivery systems, ultimately enhancing patient compliance and therapeutic outcomes. Sanofi's financial outlook for 2026 is promising, driven by robust sales of Dupixent, which exceeded €5 billion in quarterly sales. Dupixent, a monoclonal antibody used for treating atopic dermatitis and other autoimmune conditions, has been a significant revenue driver for Sanofi. The success of Dupixent reflects its effectiveness and strong market adoption, highlighting the potential of monoclonal antibodies as cornerstones of modern pharmacotherapy, particularly for chronic inflammatory diseases. Regeneron and Sanofi's Dupixent continues performing strongly with $6 billion in sales during Q2 2026—marking its largest quarterly revenue since the pandemic began—illustrating robust demand across various indications within both companies' portfolios. On the regulatory front, Alfasigma's Linerixibat (Lynavoy) has received a positive opinion from the CHMP for treating cholestatic pruritus in primary biliary cholangitis following successful Phase 3 trials. As an IBAT inhibitor targeting bile acid metabolism pathways, Lynavoy introduces a novel therapeutic approach for managing symptoms associated with this autoimmune liver disease. Meanwhile, ImmunityBio's Anktiva has gained marketing authorization in the UAE for non-muscle invasive bladder cancer and metastatic non-small cell lung cancer. Anktiva, an IL-15 cytokine-based protein therapy, exemplifies the growing interest in harnessing immune system modulators for cancer treatment. Takeda's recent decision to discontinue its nanoparticle therapy (TAK-101) for celiac disease highlights the inherent challenges in developing new therapies for autoimmune disorders. The disappointing Phase 2 results suggest that achieving immune tolerance to dietary gluten remains a significant scientific hurdle. From a strategic perspective, Sanofi's CEO has emphasized stricter go/no-go decisions during Phase 3 clinical trials amid pipeline cuts and significant impairment losses. This approach could lead to more efficient resource allocation and potentially higher success rates for late-stage drug candidates. The industry is witnessing significant transformations through strategic shifts driven by executives like Sanofi's new CEO Belen Garijo. Her vision includes reversing recent challenges faced by Sanofi by capitalizing on its strengths while addressing setbacks such as discontinuing certain late-stage clinical programs like the joint venture with Regeneron on the IL-33 candidate itepekimab. This strategic pivot mirrors broader industry trends toward optimizing late-stage pipelines to enhance competitive positioning and drive future growth. Advancements in AI-powered drug discovery continue gaining momentum through collaborations like those between GSK and Relation Therapeutics, alongside Causaly and Sage. These partnerships aim to leverage AI and machine learning technologies to accelerate drug discovery processes by integrating vast amounts of scientific literature into data analytics platforms. GlaxoSmithKline's $110 million deal with an AI biotech firm further signals increased integration between artificial intelligence technologies and pharmaceutical research efforts aimed at enhancing dataset quality thereby accelerating innovation throughout drug discovery processes. Bristol Myers Squibb faces further delays regarding its Alzheimer's psychosis treatment Cobenfy—a postponement reflecting ongoing complexities related to neurological drug development which requires overcoming high scientific hurdles alongside regulatory scrutiny. Alnylam Pharmaceuticals recently experienced a 29% drop in stock value following disappointing sales of Amvuttra and a downward revision of its ATTR franchise outlook for 2026. Such fluctuations highlight volatility within biotech investments when market expectations are not met. These collective developments reveal significant trends shaping today's pharmaceutical landscape: strategic pipeline optimization efforts alongside robust investment initiatives targeting high-demand therapeutic areas—all while leveraging technological advancements like AI integration aimed at improving R&D efficiency ultimately impacting patient care worldwide through innovative therapies addressing unmet medical needs globally.Support the show

Biogen's new data, presented at the Alzheimer's Association International Conference, supports a tau-focused approach to the intractable neurodegenerative disease; psychedelics are back in the news with more positive data from Compass Pathways and final guidance from the FDA; and the ATTR-CM space got a major shakeup with the late-stage failure of AstraZeneca and Ionis' antisense therapeutic.

On this week's episode, Sam Fazeli, Josh Schimmer, Eric Schmidt, Paul Matteis, and special guest STAT's Matt Herper open on the market, noting that despite a wobbly day for biotech, the XBI holds strong gains year-to-date. The conversation turns to FDA transparency, where the co-hosts flag that CRLs haven't been released since April and that the agency may be walking back that practice. The co-hosts debate whether the FDA is swinging too far toward leniency after Agios' mitapivat won priority review in sickle cell disease despite missing its primary endpoint last year. On AI in drug discovery, Matt Herper recaps an interview with Anthropic's CEO about a new research tool, noting that it's plausible the tool helps industry pick better targets and lower the failure rate. In M&A, Vertex's largest-ever deal takes Crinetics for $10 billion net of cash, staking a claim in the specialty rare endocrine space, while Novartis expands its ADC portfolio with Myricx for $1.1 billion upfront plus $400 million in milestones — the second European ADC deal in recent months. On data, AstraZeneca and Ionis' eplontersen trial in ATTR-CM missed its endpoint, showing no benefit or biomarker signal on top of background tafamidis; the group then discusses the readthroughs for BridgeBio, Alnylam, and Pfizer's ATTR therapies. This episode aired on July 10, 2026.

Drs. Witteles and Alexander discuss emerging data in transthyretin amyloid cardiomyopathy, highlighting durable survival benefits with long-term transthyretin-stabilizing therapy and the prognostic impact of atrial arrhythmias. They also review sex-related differences in phenotype that may contribute to under-recognition in women and emphasize earlier diagnosis with more nuanced, holistic management strategies.

Dr. Katie Krulisky and Dr. Marcus Pinto discuss the diagnosis and management of hereditary ATTR amyloidosis. Show citation:  Panrudkevich AH, Jones FJS, Shouman K, et al. Sensitivity of Nerve and Skin Biopsy and Fat Aspirate for Amyloid in Symptomatic Hereditary ATTR Amyloidosis With Peripheral Neuropathy. Neurology. 2026;106(11):e218033. doi:10.1212/WNL.0000000000218033 

On the KMOJ Morning Show, Dr. Barbara Hutchinson joins Chantel Sings to discuss ATTR-cardiac amyloidosis (ATTR-CM), a serious and progressive heart condition that is frequently misdiagnosed because its symptoms often resemble normal aging or other forms of heart failure. She explains why common warning signs—including shortness of breath, fatigue, swelling in the legs or feet, irregular heartbeat, and even conditions like carpal tunnel syndrome—should not be ignored, particularly since one in 25 Black Americans carries a genetic variant associated with hereditary ATTR-CM. Dr. Hutchinson also highlights new survey findings showing that 95% of Black women surveyed had never been screened for the condition, underscoring the need for greater awareness and earlier diagnosis. The conversation encourages listeners to have informed discussions with their health care providers about persistent symptoms and the importance of early evaluation and appropriate care.

Dr. Katie Krulisky talks with Dr. Marcus Pinto about the diagnosis and management of hereditary ATTR amyloidosis.  Read the related article in Neurology®.  Disclosures can be found at Neurology.org.  

Drs. Dasgupta and Sarswat review how ATTR cardiomyopathy remains under-recognized despite its prevalence in older patients with heart failure, emphasizing clinical red flags across cardiac and systemic manifestations. They outline a practical diagnostic pathway that prioritizes early identification using light-chain evaluation, bone scintigraphy, and genetic testing to distinguish transthyretin subtypes and initiate timely, disease-modifying therapy.

Darshan H. Brahmbhatt, Podcast Editor of JACC: Advances, discusses a recently published original research paper on Early Diagnosis of ATTR-CM by Age- and Carpal Tunnel Biopsy-Guided Screening.

Ny episode av Eventyr fra Andedammen! Vi har fått med oss poesiens nye it-boy Mats Tønjum Alfsen som er i studio og forteller om debuten sin "Svartediket". Vi har lest Samlagets erotiske fantasier "Attrå", og anbefaler Kneecaps nye plate og en utrolig film om en fotballhelt. Nevnt i ukens pod: "Svartediket" Mats Tønjum Alfsen "Altazor" Vicente Huidobro Halvor Roll (forfatter) "Salt/Land" Fredrik Parelius "Attrå. Erotiske fantasiar" Samlaget "Avkledd" Nina Brochmann "On all fours" Miranda July "Vladimir" (Serie, Netflix) "Kärlek og anarki" Serie, Netflix" "Sexistencial" Robyn "West end girl" Lily Allen "Fenian" Kneecap

Drs. Sarswat and Dasgupta discuss contemporary decision-making in treating transthyretin amyloidosis, comparing oral stabilizers with subcutaneous silencers and emphasizing earlier diagnosis, phenotype-driven therapy, and individual patient preferences. They highlight how neuropathy and autonomic dysfunction influence choice of mechanism, review practical considerations such as side-effect profiles, dosing logistics, and drug–drug interactions, and briefly touch on emerging approaches like amyloid-removing and gene-targeted therapies.

It’s a pivotal year for RNA therapeutics pioneer Ionis Pharmaceuticals. After transitioning from a partnering model to a full-ownership strategy following the 2024 approval of Tryngolza, the company is expecting an FDA decision to expand the drug into severe hypertriglyceridemia as its first non-rare-disease indication for a wholly owned product. Meanwhile, potentially game-changing phase 3 readouts are expected from Ionis’ Novartis-partnered lipoprotein(a) candidate and AstraZeneca-partnered ATTR cardiomyopathy program. In this week’s episode of "The Top Line," Fierce’s Angus Liu interviews Ionis CEO Brett Monia, Ph.D. He discusses the science of antisense oligonucleotides, the significance of the two closely watched upcoming clinical readouts, and what it takes to turn a 30-year-old discovery platform into a full-fledged commercial biotech. To learn more about the topics in this episode: Ionis slashes Tryngolza's price tag by 93% ahead of anticipated label expansion 'It’s going to be huge': The 'diabolical' molecule poised to become biotech's next gold rush AstraZeneca sets sights on 25+ blockbusters by 2030 to fuel $80B revenue ambition As questions swirl around ATTR competition, Alnylam plots path to market leadership for Amvuttra Ionis exec shares method to the Madness after 2026 Drug Name Tournament win Ionis plots FDA filing after rare neurological disease candidate posts phase 3 win See omnystudio.com/listener for privacy information.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into a fascinating array of industry shifts and scientific breakthroughs that are shaping the future of medicine and patient care. Sun Pharma's acquisition of Organon for $11.75 billion is making waves as it marks a significant expansion for the Indian pharmaceutical giant into international markets. This deal not only amplifies Sun Pharma's footprint but also integrates Organon's strong focus on women's health and biosimilars into its portfolio. It's a strategic move that underscores the increasing trend of cross-border mergers as companies seek to diversify and enhance their global presence. As the largest-ever overseas acquisition by an Indian firm, this transaction is emblematic of the growing importance of global consolidation in the pharma industry. Meanwhile, Astellas Pharma faces a mixed fiscal outlook with its prostate cancer drug Xtandi. While Xtandi experienced a modest sales increase in 2025, a projected decline for 2026 has prompted Astellas to pivot towards developing new medicines. This highlights the critical dependence on innovation within the industry to sustain growth, especially as competition intensifies. In the realm of ATTR amyloidosis treatments, Pfizer has reached a settlement with two generic manufacturers over its drug Vyndamax. This resolution is crucial in stabilizing the market amidst rising generic competition, which can significantly influence pricing and market dynamics. Such settlements are an integral part of maintaining competitive positioning in crowded therapeutic spaces. Ligand Pharmaceuticals' strategic purchase of XOMA for $739 million signals a broader trend of consolidation among biotech royalty aggregators. This acquisition aligns with Ligand's strategy to augment its portfolio through mergers and acquisitions—a recurring theme as biotech companies strive to streamline operations and expand their competitive edge. The competition within the oral GLP-1 market is intensifying, particularly with Eli Lilly's delayed launch of Foundayo inadvertently benefiting Novo Nordisk's market position. This rivalry underscores the fierce race to dominate the lucrative obesity management sector, highlighting how pivotal timing can be in gaining market share. The FDA's recent allocation of national priority vouchers to Compass Pathways, Usona Institute, and Transcend Therapeutics marks a significant regulatory shift towards embracing psychedelic therapies. These vouchers could expedite development timelines, thereby accelerating patient access to novel treatments that have traditionally been underexplored. Thermo Fisher Scientific's robust start to 2026 showcases how strategic acquisitions like that of Clario can drive growth. This reflects broader industry trends where biopharma companies leverage technological advancements to enhance capabilities in clinical trial support and data management. In gene editing news, Intellia Therapeutics is advancing towards FDA approval for its in vivo CRISPR therapy following successful Phase 3 trials. This progress heralds a new era for gene editing technologies in treating genetic disorders, underscoring their potential to revolutionize therapeutic approaches. Eli Lilly continues its aggressive acquisition strategy by purchasing Ajax Therapeutics for $2.3 billion. This acquisition focuses on next-generation JAK inhibitors for myelofibrosis, aligning with Lilly's broader goal to diversify its pipeline and maintain its leadership in innovative therapeutics. Recent studies have also shed light on how an endoscopic procedure used in Europe can aid patients on Eli Lilly's tirzepatide in maintaining weight loss post-treatment. This finding could significantly impact long-term obesity management strategies, offering new insights into sustainaSupport the show

Hoy hablamos sobre una condición poco conocida pero muy seria: la amiloidosis cardíaca por transtiretina. Una enfermedad que puede confundirse con el envejecimiento o el cansancio normal, pero que en realidad puede afectar gravemente el corazón si no se detecta a tiempo.En este episodio te explico qué es, cómo se desarrolla, cuáles son sus síntomas, por qué muchas veces se diagnostica tarde y qué opciones existen hoy en día para tratarla.La información salva vidas… y este episodio puede ayudarte a reconocer señales que no debes ignorar.✨ Si deseas producto naturales que apoyen tu equilibrio hormonal, emocional y sexual, visita mi tienda:

In this episode of the Heart podcast, Digital Media Editor, Professor James Rudd, is joined by Professor Marianna Fontana from Royal Free Hospital in London, UK. They discuss the current and future state of therapies for ATTR amyloidosis affecting the heart. If you enjoy the show, please leave us a supportive review wherever you get your podcasts. It helps us to reach more people - thanks!   Link to published paper: https://heart.bmj.com/content/112/3/129

In this episode of JACC This Week, Dr. Harlan M. Krumholz and Dr. Carolyn S.P. Lam discuss a dedicated issue of JACC focused on cardiac amyloidosis—one of the fastest-evolving areas in cardiovascular medicine. They explore new evidence highlighting significant delays in diagnosing ATTR cardiomyopathy, the early divergence of mortality benefit with timely treatment, and why time to diagnosis is no longer a neutral factor. The conversation also examines secondary analyses from major clinical trials, practical guidance for amyloidosis evaluation and management, and Dr. Krumholz's Editor's Page on "computable diagnosis" as a moral imperative. This episode places emerging science in clinical context, emphasizing urgency, equity, and how clinicians should be thinking differently about diagnosis, staging, and access to therapy in amyloid heart disease. Read Full issue here: https://www.jacc.org/toc/jacc/87/5 Keywords: cardiac amyloidosis, amyloid heart disease, ATTR cardiomyopathy, computable diagnosis

With Felix Lindberg, Karolinska Institutet & Södersjukhuset Hospital, Stockholm – Sweden and Marianna Fontana, National Amyloidosis Center, University College London, London – UK. In this episode, Felix Lindberg and Marianna Fontana discuss the evolving landscape in the treatment of cardiac amyloidosis. After exploring the pathophysiology of cardiac AL and ATTR amyloidosis and recent years' insights about the epidemiology of these conditions, they focus on the transformation that has occurred in the therapeutic options for cardiac ATTR amyloidosis. The conversation addresses the key mechanistic differences and trial data on stabilizers and silencers, how to approach patient selection for these therapies, as well as emerging therapies including anti-amyloid antibodies. The discussion also highlights the importance of early diagnosis and key red flags that should elicit suspicion of cardiac amyloidosis. Suggested reading: Vutisiran in patients with transthyretin amyloidosis with cardiomyopathy. Fontana M, et al. N Engl J Med. 2025. Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy. Maurer M, et al. N Engl J Med. 2018. Efficacy and safety of acoramidis in tranthyretin amyloid cardiomyopathy. Gillmore JD, et al. N Engl J Med. 2024. CRISPR-Cas9 gene editing with nexiguran ziclumeran for ATTR cardiomyopathy. Fontana M, et al. N Engl J Med. 2024. Conventional heart failure therapy in cardiac ATTR amyloidosis. Ioannou A, et al. Eur Heart J. 2023. Nonbiopsy diagnosis of cardiac transthyretin amyloidosis. Gillmore JD, et al. Circulation. 2016. Current landscape of therapies for transthyretin amyloid cardiomyopathy. Griffin JM, et al. JACC Heart Fail. 2025. Transthyretin amyloid cardiomyopathy: from cause to novel treatments. Fontana M, et al. Eur Heart J. 2026. Diagnosis and treatment of cardiac amyloidosis: a position statement of the ESC Working Group on Myocardial and Pericardial Diseases. Garcia-Pavia P, et al. Eur Heart J. 2021. This 2026 HFA Cardio Talk podcast series is supported by Bayer in the form of unrestricted financial support. The discussion has not been influenced in any way by its sponsor.

AI Detection of Cardiac Amyloidosis   Guest: Patricia Pellikka, M.D. Host: Malcolm Bell, M.D.   Dr. Patricia Pellikka dives into the AI model she helped build. Working with an AI echo company, Ultromics Ltd., her team built an AI echo model to detect cardiac amyloidosis. The model works from an apical 4 chamber videoclip and was approved by the FDA via the breakthrough pathway. They have validated it internationally in a population with 22% prevalence of cardiac amyloidosis and it had a sensitivity of 85%, specificity 93%, PPV 78%, NPV 96%, and AUC 0.93.  The performance of the model was consistent across cardiac amyloid types; among AL, wild type ATTR, and hereditary ATTR, the sensitivity was 84%, 85%, and 86%, respectively.  The performance of the model was also superior to previously validated scores which use clinical and echocardiographic variables for detecting cardiac amyloidosis. The results of the study were published online in the European Heart Journal in July.   Topics Discussed: You have built an AI echo model to detect cardiac amyloidosis.  How did you get into this? Tell us about the model that you built. How did you validate it? How does the model compare with other screening methods for detecting cardiac amyloidosis? What are the next steps for your research?   Connect with Mayo Clinic's Cardiovascular Continuing Medical Education online at https://cveducation.mayo.edu or on Twitter @MayoClinicCV and @MayoCVservices. LinkedIn: Mayo Clinic Cardiovascular Services Cardiovascular Education App: The Mayo Clinic Cardiovascular CME App is an innovative educational platform that features cardiology-focused continuing medical education wherever and whenever you need it. Use this app to access other free content and browse upcoming courses. Download it for free in Apple or Google stores today! No CME credit offered for this episode.   Podcast episode transcript found here.

Do you know how to recognize ATTR earlier, navigate complex diagnostics, and apply the latest therapeutic innovations?   Credit available for this activity expires: 12/23/2026 Earn Credit / Learning Objectives & Disclosures: https://www.medscape.org/viewarticle/real-time-insights-diagnosis-and-management-attr-genetic-2025a1000yeo?ecd=bdc_podcast_libsyn_mscpedu

Erwan Donal, MD, PhD / Nina Ajmone Marsan, MD, PhD - At the Cutting Edge of ATTR-CM: How Can We Leverage Advances in Multimodality Cardiac Imaging and Artificial Intelligence to Modernise Diagnosis and Monitoring?

Erwan Donal, MD, PhD / Nina Ajmone Marsan, MD, PhD - At the Cutting Edge of ATTR-CM: How Can We Leverage Advances in Multimodality Cardiac Imaging and Artificial Intelligence to Modernise Diagnosis and Monitoring?

Erwan Donal, MD, PhD / Nina Ajmone Marsan, MD, PhD - At the Cutting Edge of ATTR-CM: How Can We Leverage Advances in Multimodality Cardiac Imaging and Artificial Intelligence to Modernise Diagnosis and Monitoring?

Francesco Cappelli, MD, PhD - Meaningful Data From Madrid: Expanding Our Understanding of Phase 3s and OLEs in ATTR-CM

Francesco Cappelli, MD, PhD - Meaningful Data From Madrid: Expanding Our Understanding of Phase 3s and OLEs in ATTR-CM

Im Binärgewitter-Talk #370 stolpern wir gemeinsam durch die glitzernde Tech-Welt – von Linux-Liebeserklärungen bis Mac-Mimimi. Unser Gast erklärt uns, warum Stromnetze spannender sind als jede Netflix-Serie, während Cloud-Dienste reihenweise „Tote der Woche“ melden. Zwischendurch philosophieren wir über Kubernetes, KI-Hacking und ob Gateway-API wirklich das neue heiße Ding ist. Zum Schluss gibt's Zukunftsvisionen zu E-Mobilität, Smart Homes und Mini-Windrädern – Tech-Chaos zum Mitlachen garantiert! Toter der Woche graveyard has a new logo Neato Cloud Services MinIO Ingress NGINX Retirement Externe Facebook “like” und “comment” buttons Exotische Debian Ports Plain HTTP in Chrome Lennarts Blog Untoter der Woche Linux-Konsole: Valve kündigt neue Steam Machine an Steam Hardware Announcement AI der Woche AI Darwin Awards Securevibes Volkwagen for Unit Tests Where’s the Shovelware? Why AI Coding Claims Don’t Add Up Anthropic: AI Espionage Researchers Question claim AI slop attacks on the curl project (video) Blog Post von Daniel Stenberg AI Song an der Spitze der Charts (in den USA) Human Music (video) Cometjacking attack Unseeable prompt injections in Comet and other AI browsers AI World Clocks News Fedora Linux 43 Meta wants to read your DMs Operaton has reached 1.0 — Camunda 7.0 CE repo has been archived FreeBSD shortly before 15.0: Trust is good, reproducibility is better FreeBSD now builds reproducibly and without root privilege PS5 Funktionierender User + Kernel Exploit Affinity's new design platform combines everything into one app Ausbruch aus Dockercontainer Themen eAuto laden und Energienetze (follow up zur FrosCon Folge) Wikipedia: Grobe Struktur eines Stromnetzes Frische News Schuko für PV Maus: Pumpspeicherwerk DLF Forschung Aktuell — Podcast: Wasserstofferzeugung Wikipedia: Hochspannungs-Gleichstrom-Übertragung Wikipedia: Karte Offshore-Windparks in der Deutschen Bucht Wikipedia: Kleinwindkraftanlage 3D-Druck der Woche I Broke the Sound Barrier with a 3D Printed Rocket! (video) C-Hook Battery Cover Mimimi der Woche Anycubic Slicer Next für Linux nur mit “execute Shellscript from internet” welches CN schriftzeichen als Meldungen ausgibt die Installationsziele auf Ubuntu Only einschränkt im Endeffekt doch nur eine Paket-Source einträgt und via apt ein Paket installiert NixOS static ip let ext-if = "et0"; external-mac = "00:11:22:33:44:55"; external-ip6 = "2a01::2342"; external-netmask6 = "64"; in { services.udev.extraRules = '' SUBSYSTEM=="net", ATTR{address}=="${external-mac}", NAME="${ext-if}" ''; networking = { enableIPv6 = true; nat.enableIPv6 = true; interfaces."${ext-if}" = { useDHCP = true; ipv6.addresses = [{ address = external-ip6; prefixLength = external-netmask6; }]; }; defaultGateway6 = { address = external-gw6; interface = ext-if; }; nameservers = [ "1.1.1.1" ]; }; } Ab-er Finger macht kein Touch Lesefoo OpenSource Alternativen zu Cloudflare Picks thingino Severance S02 Kittysplit seized.fyi Tooling https://volta.sh/ https://github.com/Schniz/fnm https://mise.jdx.dev/ Fwupd 2.0.16 Released Mit OSS Termine buchen beim Arzt Bahnstationen in 3D-Karte

Emer Joyce, MB BCh BAO, PhD - Staying Ahead of the Curve in ATTR-CM: Adapting and Advancing Our Approach to Decision-Making as the Treatment Landscape Evolves

Emer Joyce, MB BCh BAO, PhD - Staying Ahead of the Curve in ATTR-CM: Adapting and Advancing Our Approach to Decision-Making as the Treatment Landscape Evolves

Emer Joyce, MB BCh BAO, PhD - Staying Ahead of the Curve in ATTR-CM: Adapting and Advancing Our Approach to Decision-Making as the Treatment Landscape Evolves

This episode covers: Cardiology This Week: A concise summary of recent studies Arrhythmias in cardiac amyloidosis Taking the 'O' out of HOCM: managing LVOT obstruction Snapshots Host: Susanna Price Guests: Carlos Aguiar, Stephanie Schwarting, Ahmad Masri Want to watch that episode? Go to: https://esc365.escardio.org/event/2176 Want to watch that extended interview on Arrhythmias in Cardiac Amyloidosis? Go to: https://esc365.escardio.org/event/2176?resource=interview Disclaimer: ESC TV Today is supported by Bristol Myers Squibb and Novartis through an independent funding. The programme has not been influenced in any way by its funding partners. This programme is intended for health care professionals only and is to be used for educational purposes. The European Society of Cardiology (ESC) does not aim to promote medicinal products nor devices. Any views or opinions expressed are the presenters' own and do not reflect the views of the ESC. The ESC is not liable for any translated content of this video. The English language always prevails. Declarations of interests: Stephan Achenbach, Yasmina Bououdina, Nicolle Kraenkel and Susanna Price have declared to have no potential conflicts of interest to report. Carlos Aguiar has declared to have potential conflicts of interest to report: personal fees for consultancy and/or speaker fees from Abbott, AbbVie, Alnylam, Amgen, AstraZeneca, Bayer, BiAL, Boehringer-Ingelheim, Daiichi-Sankyo, Ferrer, Gilead, GSK, Lilly, Novartis, Pfizer, Sanofi, Servier, Takeda, Tecnimede. John-Paul Carpenter has declared to have potential conflicts of interest to report: stockholder Mycardium AI. Davide Capodanno has declared to have potential conflicts of interest to report: Bristol Myers Squibb, Daiichi Sankyo, Sanofi Aventis, Novo Nordisk, Terumo. Konstantinos Koskinas has declared to have potential conflicts of interest to report: honoraria from MSD, Daiichi Sankyo, Sanofi. Ahmad Masri has declared to have potential conflicts of interest to report: research grants from Pfizer, Ionis, Attralus, Cytokinetics and Janssen. Consulting fees from Cytokinetics, BMS, BridgeBio, Pfizer, Ionis, Lexicon, Attralus, Alnylam, Haya, Alexion, Akros, Edgewise, Rocket, Lexeo, Prothena, BioMarin, AstraZeneca, Avidity, Neurimmune, and Tenaya. Steffen Petersen has declared to have potential conflicts of interest to report: consultancy for Circle Cardiovascular Imaging Inc. Calgary, Alberta, Canada. Stephanie Schwarting has declared to have potential conflicts of interest to report: advisory board for Alnylam, Bayer, Pfizer; principal investigator in trials sponsored by Alexion, Novo Nordisk and Intellia. Emma Svennberg has declared to have potential conflicts of interest to report: Abbott, Astra Zeneca, Bayer, Bristol-Myers, Squibb-Pfizer, Johnson & Johnson.

Host: Susanna Price Guest: Stephanie Schwarting Want to watch the episode? Go to: https://esc365.escardio.org/event/2176 Want to watch the extended interview on Arrhythmias in Cardiac Amyloidosis? Go to: https://esc365.escardio.org/event/2176?resource=interview Disclaimer: ESC TV Today is supported by Bristol Myers Squibb and Novartis through an independent funding. The programme has not been influenced in any way by its funding partners. This programme is intended for health care professionals only and is to be used for educational purposes. The European Society of Cardiology (ESC) does not aim to promote medicinal products nor devices. Any views or opinions expressed are the presenters' own and do not reflect the views of the ESC. The ESC is not liable for any translated content of this video. The English language always prevails. Declarations of interests:  Stephan Achenbach, Yasmina Bououdina, Nicolle Kraenkel and Susanna Price have declared to have no potential conflicts of interest to report. Carlos Aguiar has declared to have potential conflicts of interest to report: personal fees for consultancy and/or speaker fees from Abbott, AbbVie, Alnylam, Amgen, AstraZeneca, Bayer, BiAL, Boehringer-Ingelheim, Daiichi-Sankyo, Ferrer, Gilead, GSK, Lilly, Novartis, Pfizer, Sanofi, Servier, Takeda, Tecnimede. John-Paul Carpenter has declared to have potential conflicts of interest to report: stockholder Mycardium AI. Davide Capodanno has declared to have potential conflicts of interest to report: Bristol Myers Squibb, Daiichi Sankyo, Sanofi Aventis, Novo Nordisk, Terumo. Konstantinos Koskinas has declared to have potential conflicts of interest to report: honoraria from MSD, Daiichi Sankyo, Sanofi. Steffen Petersen has declared to have potential conflicts of interest to report: consultancy for Circle Cardiovascular Imaging Inc. Calgary, Alberta, Canada.  Stephanie Schwarting has declared to have potential conflicts of interest to report: advisory board for Alnylam, Bayer, Pfizer; principal investigator in trials sponsored by Alexion, Novo Nordisk and Intellia. Emma Svennberg has declared to have potential conflicts of interest to report: Abbott, Astra Zeneca, Bayer, Bristol-Myers, Squibb-Pfizer, Johnson & Johnson.

What are the early signs of cardiac amyloidosis? Dr Alexander reviews the disproportionate burden of disease, early signs, and treatment options. Credit available for this activity expires: 9/24/26 Earn Credit / Learning Objectives & Disclosures: https://www.medscape.org/viewarticle/recognizing-and-managing-variant-attr-amyloidosis-focus-2025a1000p21?ecd=bdc_podcast_libsyn_mscpedu

This week, Dr. Kahn reports on a new Mayo Clinic study on the relationship between chronic insomnia and accelerated brain aging in a group of study subjects who had advanced brain testing over time. He suggests 20 tips for better sleep and a bonus 21st: cognitive behavioral therapy for insomnia (CBT-I). Additional topics include heart disease remaining the #1 cause of death, TMAO and cognitive impairment, ATTR-cardiomyopathy, childhood blood pressure and future heart disease, niacin and optimal liver health, and the relationship between heart diseases like atrial fibrillation and cognitive decline. Thanks to igennus.com and the discount code DrKahn.  Dr. Kahn will be leading a group Fasting Mimicking Diet starting 10/5/25. Order your PROLON kit (Next Gen) at www.prolonlife.com/drkahn.

This week, Dr. Joel Kahn shares the case of a patient at the Kahn Center diagnosed with ATTR cardiomyopathy, a serious disorder in which a protein (amyloid) infiltrates the heart and can be fatal. It is often associated with carpal tunnel syndrome, neuropathy, and spinal stenosis. Fortunately, exciting new therapies are now available. Other topics in this episode include the latest cholesterol guidelines, niacin and mood, Galleri cancer screening, “inflammaging” in native cultures, beta-blockers after heart attack, menopause and aging, and inflammation in women. Thanks to Igennus.com — use discount code DrKahn.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/YFD865. CME credit will be available until August 26, 2026.Expert Perspectives on Novel Approaches to Therapy for ATTR Cardiac Amyloidosis In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an educational grant from Alnylam Pharmaceuticals, Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/YFD865. CME credit will be available until August 26, 2026.Expert Perspectives on Novel Approaches to Therapy for ATTR Cardiac Amyloidosis In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an educational grant from Alnylam Pharmaceuticals, Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/YFD865. CME credit will be available until August 26, 2026.Expert Perspectives on Novel Approaches to Therapy for ATTR Cardiac Amyloidosis In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an educational grant from Alnylam Pharmaceuticals, Inc.Disclosure information is available at the beginning of the video presentation.

Darshan H. Brahmbhatt, Podcast Editor of JACC: Advances, discusses a recently published original research paper on Benefits of Patisiran on Functional Capacity in ATTR Cardiac Amyloidosis: Post Hoc Analysis of APOLLO-B.

CardioNerds (Drs. Rick Ferraro and Georgia Vasilakis Tsatiris) discuss ATTR cardiac amyloidosis with expert Dr. Justin Grodin. This episode is a must-listen for all who want to know how to diagnose and treat ATTR with current available therapies, as well as management of concomitant diseases through a multidisciplinary approach. We take a deep dive into the importance of genetic testing, not only for patients and families, but also for gene-specific therapies on the horizon. Dr. Grodin draws us a roadmap, guiding us through new experimental therapies that may reverse the amyloidosis disease process once and for all.  Audio editing by CardioNerds academy intern, Christiana Dangas. This episode was developed in collaboration with the American Society of Preventive Cardiology and supported by an educational grant from BridgeBio.  Enjoy this Circulation Paths to Discovery article to learn more about the CardioNerds mission and journey.  US Cardiology Review is now the official journal of CardioNerds! Submit your manuscripts here.  CardioNerds Cardiac Amyloid PageCardioNerds Episode Page Pearls: You must THINK about your patient having amyloid to recognize the pattern and make the diagnosis. Start with a routine ECG and TTE, and look for a disproportionately large heart muscle with relatively low voltages on the ECG.  Before you diagnose ATTR amyloidosis, AL amyloidosis must be ruled out (or ruled in) with serum light chains, serum/urine immunofixation, and/or tissue biopsy.  Genetic testing is standard of care for all patients and families with ATTR amyloidosis, and the future is promising for gene-specific treatments. Current FDA-approved treatments for TTR amyloidosis are TTR stabilizers and TTR silencers, but TTR fibril-depleting agents are on their way.  Early diagnosis of ATTR affords patients maximal benefit from current amyloidosis therapies.   TTR amyloidosis patients require a multidisciplinary approach for success, given the high number of concomitant diseases with cardiomyopathy.  Notes: Notes: Notes drafted by Dr. Georgia Vasilakis Tsatiris.  What makes you most suspicious of a diagnosis of cardiac amyloidosis from the typical heart failure patient?  You must have a strong index of suspicion, meaning you THINK that the patient could have cardiac amyloidosis, to consider it diagnostically. Some characteristics or “red flags” to not miss:   Disproportionately thick heart muscle with a relatively low voltages on EKG   Bilateral carpal tunnel syndrome – estimated that 1 in 10 people >65 years old will have amyloidosis   Previously tolerated antihypertensive medications  Atraumatic biceps tendon rupture   Bilateral carpal tunnel syndrome  Spinal stenosis   Concomitant with other diseases: HFpEF, low-flow low-gradient aortic stenosis  How would you work up a patient for cardiac amyloidosis?   Start with a routine ECG (looking for disproportionally low voltage) and routine TTE (looking for thick heart muscle)  CBC, serum chemistries, hepatic function panel, NT proBNP, and troponin levels  NOTE: It is critical to differentiate between amyloid light chain (AL amyloidosis) and transthyretin ATTR amyloidosis, as both make up 95-99% of amyloidosis cases.   Obtain serum free light chains, serum & urine electrophoresis, and serum & urine immunofixation to rule out AL amyloidosis. (See table below)  AL Amyloidosis ATTR Amyloidosis  → Positive serum free light chains and immunofixation (Abnormal M protein) → Tissue biopsy (endomyocardial, fat pad) to confirm diagnosis → Negative serum free light chains and immunofixation (ruled out AL amyloidosis) → Cardiac scintigraphy (Technetium pyrophosphate with SPECT imaging)  What treatment options do we have to offer now for ATTR CM, and how has this compared to prior years?   Before 2019, treatment options were limited outside of cardiac tr...

No episódio de hoje do check-up semanal, nosso editor-chefe médico, Ronaldo Gismondi, traz os principais destaques do mês sobre cardiologia abordados no Portal Afya.Confira os temas do episódio de hoje sobre cardiologia: Eficácia do acoramidis no tratamento da amiloidose ATTR; Biomarcadores hepáticos têm valor prognóstico na ICFEP?; Troponina para estimativa de risco cardiovascular; Lipoproteína (a): Consenso Internacional de Bruxelas aborda o tema; Proteção cerebral durante a TAVI. Aperte o play e ouça agora os destaques!

oin i3 Health for a special live webinar, Increasing Detection and Improving Outcomes in Hereditary Transthyretin Amyloid Cardiomyopathy, being held on Wednesday, July 30 at 5:00 pm ET. In this exclusive event, Dr. Mathew Maurer, the Arnold and Arlene Goldstein Professor of Cardiology at Columbia University Irving Medical Center, will share his expertise and latest insights in the treatment of hereditary ATTR-CM. In this interview, Dr. Maurer shares a preview of the topics he is excited to highlight during the webinar, including signs and symptoms to aid in early diagnosis, advances in genetic testing and new diagnostic modalities, novel therapies that are improving outcomes, and the role of interdisciplinary collaboration. Don't miss this opportunity to learn from one of the leading voices in hereditary ATTR-CM care. Register at the link below or scan the QR code in the video to secure your spot. We look forward to seeing you! Register Here: bit.ly/3UgV7nF

Drs. Rick Ferraro and Sneha Nandy discuss ‘Diagnosis of ATTR Cardiac Amyloidosis' with Dr. Venkatesh Murthy.  In this episode, we explore the diagnosis of ATTR cardiac amyloidosis, a condition once considered rare but now increasingly recognized due to advances in imaging and the availability of effective therapies. Dr. Venkatesh Murthy, a leader in multimodality imaging, discusses key clinical and laboratory features that should raise suspicion for the disease. We also examine the role of nuclear imaging and genetic testing in confirming the diagnosis, as well as the importance of early detection. Tune in for expert insights on navigating this challenging diagnosis and look out for our next episode on treatment approaches for cardiac amyloidosis! Audio editing for this episode was performed by CardioNerds Intern, Julia Marques Fernandes. Enjoy this Circulation Paths to Discovery article to learn more about the CardioNerds mission and journey.  US Cardiology Review is now the official journal of CardioNerds! Submit your manuscripts here.  CardioNerds Cardiac Amyloid PageCardioNerds Episode Page Pearls: - Diagnosis of Transthyretin amyloid cardiomyopathy 1. Recognizing the Red Flags – ATTR cardiac amyloidosis often presents with subtle but telling signs, such as bilateral carpal tunnel syndrome, low-voltage ECG, and a history of lumbar spinal stenosis or biceps tendon rupture. If you see these features in a patient with heart failure symptoms, think amyloidosis!    2. “Vanilla Ice Cream with a Cherry on Top” – On strain echocardiography, apical sparing is a classic pattern for cardiac amyloidosis. While helpful, it's not foolproof—multimodal imaging and clinical suspicion are key!   3. Nuclear Imaging is a Game-Changer – When suspicion for cardiac amyloidosis is high à a positive PYP scan with SPECT imaging (grade 2 or 3 myocardial uptake) in the absence of monoclonal protein (ruled out by SPEP, UPEP, and free light chains) is diagnostic for ATTR amyloidosis—no biopsy needed!   4. Wild-Type vs. Hereditary? Know the Clues – Older patients (70+) are more likely to have wild-type ATTR, while younger patients (40s-60s), especially those with neuropathy and a family history of heart failure, should raise suspicion for hereditary ATTR. Genetic testing is crucial for distinguishing between the two. Note that some ATTR variants may predispose to a false negative PYP scan!  5. Missing Amyloidosis = Missed Opportunity – With multiple disease-modifying therapies now available, early diagnosis is critical. If you suspect cardiac amyloidosis, don't delay the workup—early treatment improves outcomes!   Notes - Diagnosis of Transthyretin amyloid cardiomyopathy What clinical features should raise suspicion for ATTR cardiac amyloidosis?   ATTR cardiac amyloidosis is underdiagnosed because symptoms overlap with other forms of heart failure.   Red flags include bilateral carpal tunnel syndrome (often years before cardiac symptoms), low-voltage ECG despite increased LV wall thickness, heart failure with preserved ejection fraction (HFpEF) with a restrictive pattern, and history of lumbar spinal stenosis, biceps tendon rupture, and/or peripheral neuropathy, including possible autonomic dysfunction (e.g., orthostatic hypotension).  Remember: If an older patient presents with heart failure and unexplained symptoms like neuropathy or musculoskeletal issues, think amyloidosis!   What is the differential diagnosis for a thick left ventricle (LVH) and how does ATTR amyloidosis fit into it?    Hypertension: Most common cause of LVH, typically with a history of uncontrolled high blood pressure.   Aortic stenosis: May present with concentric LVH.   Hypertrophic cardiomyopathy (HCM): Genetic disorder typically presenting with asymmetric LVH, especially in younger patients.   Infiltrative cardiomyopathy: Often due to amyloidosis, sarcoidosis,

On this episode of Longevity by Design, Dr. Gil Blander sits down with Dr. Karl Pfleger, aging biotechnology investor and creator of AgingBiotech.info, to dissect the rapidly evolving field of aging therapeutics. Karl breaks down the sector into two main categories: interventions that slow aging rates versus rejuvenation strategies that repair accumulated damage.Karl highlights the most promising areas in his investment portfolio, including senolytics for clearing senescent cells and epigenetic reprogramming technologies. He explains why current aging clocks aren't ready for clinical use and discusses the limitations of popular interventions like GLP-1 drugs and fasting protocols. The conversation reveals that 14 Phase 3 clinical trials are currently testing core aging therapeutics.The discussion covers recent FDA approvals for ATTR treatments and stem cell therapies, signaling the field's maturation. Karl emphasizes that meaningful life extension requires a "divide and conquer" strategy, targeting multiple aging mechanisms simultaneously rather than relying on single interventions. Despite exciting therapeutic developments, he maintains that optimizing diet, exercise, and sleep remains the most impactful strategy for extending healthspan today.Guest-at-a-Glance

Marianna Fontana, MD, PhD / Julian Gillmore, MD, PhD, FRCP, FRCPath - Identifying Outcomes That Matter in ATTR-CM: Critical Considerations in the Interpretation of Contemporary Clinical Trials

JACC: Associate Editor Michelle M. Kittleson, MD, PhD, FACC, and JACC: CardioOncology Deputy Editor Ronald Witteles, MD, FACC, discuss the current findings on the impact of vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). This study on HELIOS-B comprises details on cardiovascular (CV) events and provides updated mortality analyses from a later data cut than the primary analysis. Risk of ACM and CV events, including CV hospitalizations and heart failure events, was reduced with vutrisiran versus placebo in the overall population, with consistent trends regardless of baseline tafamidis use. These findings reinforce the balanced benefit on mortality and CV events demonstrated in the primary analysis of HELIOS-B.

Watch here for a video interview with JACC Associate Editor Michelle Kittleson, MD, FACC, and author Mathew S. Muarer, MD, FACC, as they discuss Dr. Maurer's study published in JACC and presented at ACC.25. This exploratory analysis of HELIOS-B assessed the efficacy of vutrisiran versus placebo in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) by subgroups of baseline heart failure severity (primarily by NYHA class and NT-proBNP levels). Vutrisiran showed evidence of benefit vs placebo on mortality, cardiovascular events, functional capacity, quality of life, and cardiac biomarkers across the range of baseline disease severities in patients enrolled in HELIOS-B, with greatest benefit observed in patients with earlier, less severe disease.

In this episode, Dr. Valentin Fuster highlights a groundbreaking study on acoramides, a novel treatment for transthyretin amyloid cardiomyopathy (ATTR), which shows significant reductions in mortality and cardiovascular hospitalizations compared to a placebo. Experts discuss the clinical implications, comparing acoramides to the previously approved tafamidis, noting exciting advancements in treatments for a once-untreatable disease.

CardioNerds Cardiac Amyloidosis Series Chair Dr. Rick Ferraro and Episode Lead Dr. Anna Radakrishnan discuss the biology of transthyretin amyloid cardiomyopathy (ATTR-CM ) with Dr. Daniel Judge.  Notes were drafted by Dr. Anna Radakrishnan. The audio was engineered by student Dr. Julia Marques.  This episode provides a comprehensive overview of transthyretin (ATTR) cardiac amyloidosis, a complex and rapidly evolving disease process. The discussion covers the key red flags for cardiac amyloidosis, the diagnostic pathway, and the implications of hereditary versus wild-type ATTR. Importantly, the episode delves into the current and emerging therapies for ATTR, including stabilizers, gene silencers, and promising treatments like CRISPR-Cas9 and antibody-based approaches. Dr. Judge shares his insights and excitement about the rapidly advancing field, highlighting the need for early diagnosis and the potential to improve long-term outcomes for patients with this condition.  Enjoy this Circulation Paths to Discovery article to learn more about the CardioNerds mission and journey.  US Cardiology Review is now the official journal of CardioNerds! Submit your manuscripts here.  CardioNerds Cardiac Amyloid PageCardioNerds Episode Page Pearls: - Biology of Transthyretin amyloid cardiomyopathy Maintain a high index of suspicion! Look for subtle (yet telling) signs like ventricular hypertrophy, discordant EKG findings, bilateral carpal tunnel syndrome, and spontaneous biceps tendon rupture.  Utilize the right diagnostic tests. Endomyocardial biopsy remains the gold standard, but non-invasive tools like PYP scan with SPECT imaging and genetic testing are essential for accurate diagnosis.  Differentiating hereditary from wild-type ATTR is critical, as genetic forms may have a more aggressive course and familial implications.  Early diagnosis and intervention significantly improve prognosis, making vigilance in screening and prompt treatment initiation essential.  The future is now! Cutting-edge therapies are transforming the treatment landscape, including TTR stabilizers, gene silencers, and emerging technologies like CRISPR-Cas9 and antibody-based treatments.  Notes - Biology of Transthyretin amyloid cardiomyopathy What is transthyretin amyloid (aTTR) and how is it derived?  Transthyretin (TTR) is a transport protein primarily synthesized by the liver, responsible for carrying thyroid hormones (thyroxine) and retinol (vitamin A) in the blood. It circulates as a tetramer, composed of four identical monomers, which is essential for its stability and function.  In transthyretin amyloid (ATTR) amyloidosis, the TTR protein becomes unstable, leading to its dissociation into monomers. These monomers misfold and aggregate into insoluble amyloid fibrils, which deposit extracellularly in tissues such as the heart, nerves, and gastrointestinal tract. This progressive amyloid deposition leads to organ dysfunction, including restrictive cardiomyopathy and neuropathy.  There are two main forms of ATTR amyloidosis: hereditary (variant) and wild-type (senile) ATTR.  Hereditary ATTR (ATTRv) is caused by mutations in the TTR gene. These mutations destabilize the TTR tetramer, making it more prone to dissociation. This increases misfolding and amyloid fibril formation, resulting in systemic amyloid deposition.   Wild-type ATTR (ATTRwt) occurs without genetic mutations and is primarily age-related. Over time, even normal TTR tetramers can become unstable, leading to gradual misfolding and amyloid deposition, particularly in the heart. ATTRwt is a common but often underdiagnosed cause of heart failure with preserved ejection fraction (HFpEF) in elderly individuals.  How does aTTR lead to deleterious effects in the heart and other organ systems?    Transthyretin amyloidosis leads to organ dysfunction through the deposition of misfolded TTR protein as amyloid fib...

Join CardioNerds Heart Failure Section Chair Dr. Jenna Skowronski, episode lead Dr. Apoorva Gangavelli, and expert faculty Dr. Ronald Witteles as they discuss the Nex-Z trial. This was a phase 1, open-label trial investigating nex-z, a CRISPR-Cas9-based treatment, in 36 patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The primary objectives were aimed at studying the safety and pharmacodynamics of this novel gene-based treatment modality. This episode dives into the nuances of the data, future directions for investigation, and future clinical implications. CardioNerds Journal Club PageCardioNerds Episode PageCardioNerds AcademyCardionerds Healy Honor Roll CardioNerds Journal ClubSubscribe to The Heartbeat Newsletter!Check out CardioNerds SWAG!Become a CardioNerds Patron! References - The Nex-Z Trial Fontana, M., Solomon, S. D., Kachadourian, J., Walsh, L., Rocha, R., Lebwohl, D., Smith, D., Täubel, J., Gane, E. J., Pilebro, B., Adams, D., Razvi, Y., Olbertz, J., Haagensen, A., Zhu, P., Xu, Y., Leung, A., Sonderfan, A., Gutstein, D. E., & Gillmore, J. D. (2024). CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy. The New England Journal of Medicine. https://doi.org/10.1056/NEJMoa2412309

Featuring articles on advanced melanoma, atrial fibrillation, ATTR amyloidosis, and bladder cancer; a review article on cervical cancer; a Clinical Problem-Solving describing when blurry vision clouds the bigger picture; Medicine and Society's on transitional justice and on rethinking access to HIV medicines; and Perspectives on hepatitis C therapies, on when diversity goals meet multiregional trials, and on miles to go and nowhere to sleep.