Interview with the Innovators

Unraveling the Complexities of Transthyretin Amyloidosis (ATTR)

Amyloidosis News has launched a 9-part series of video clips with top experts in the field of amyloidosis research using its “Interview with the Innovators” platform. The series focuses on the role of a protein called transthyretin, or TTR in normal physiology, the pathophysiology of TTR amyloidosis (ATTR), the potential clinical implications of novel disease-specific therapies, and the role of serum TTR as a biomarker.

Advances in our knowledge of how complex diseases such as amyloidosis develop and progress over time are fundamental in the development of better therapies. Over the past few years, key information has emerged on the crucial role that TTR plays in this disease. The studies that led to our enhanced understanding of ATTR as a protein misfolding disorder were conducted across many disciplines, such as genetics and genomics (ie, phenotype to gene), reverse genetics (ie, gene to phenotype), biochemistry, molecular biology, and translational science (ie, studies on the formation of abnormal deposition of amyloid in the organs and tissues of affected patients). All of the above information contributed to rational drug design approaches that directly target the purported mechanism of the disease, or MOD in amyloidosis, leading to clinical studies with novel disease-specific medicines.

The Role of Transthyretin (TTR) in Normal Human Physiology
The first video in the 9-part “Interview with the Innovators” series is titled “The Role of Transthyretin (TTR) in Normal Human Physiology.” In this video clip, Joel N. Buxbaum, MD, Professor Emeritus, Molecular Medicine, The Scripps Research Institute, and Chief Medical Officer (consulting), Protego Biopharma, discusses the principal, as well as, less defined mechanisms underlying the physiologic functions of TTR in healthy individuals. These learnings are key for the optimal understanding of disease states associated with abnormalities in TTR structure and function. Read More ›

Transthyretin (TTR) amyloidosis: Disease Definition and Key Clinical Features
Ola Akinboboye, MD, MPH, MBA, Clinical Professor, Cardiology, Donald and Barbara Zucker School of Medicine at Hofstra University/Northwell Health and Medical Director, Laurelton Heart Specialists PC (Rosedale, NY) describes the clinical manifestations of wild-type and hereditary or variant transthyretin amyloidosis (ATTR) and some common mutational drivers of pathogenic phenotypes. Read More ›

Transthyretin (TTR) amyloidosis: Main Types and Disease Trajectory Over Time
Ola Akinboboye, MD, MPH, MBA, Clinical Professor, Cardiology, Donald and Barbara Zucker School of Medicine at Hofstra University/Northwell Health and Medical Director, Laurelton Heart Specialists PC (Rosedale, NY) discusses the pathophysiologic features of TTR amyloidosis (ATTR) as a disease entity and introduces the two types of ATTR, wild-type and hereditary or variant. Read More ›

Complexities of Transthyretin Amyloid Disposition
Joel N. Buxbaum, MD, Professor Emeritus, Molecular Medicine, The Scripps Research Institute, and Chief Medical Officer (consulting), Protego Biopharma, explores the mechanisms of organ damage in TTR amyloidosis (ATTR). This important information focuses on the biophysical and biochemical underpinnings of misfolded transthyretin (TTR) leading to tissue dysfunction in this disease. Read More ›

Transthyretin (TTR) Amyloidosis: Current Diagnostic Approaches in the Clinic
Justin L. Grodin, MD, MPH, Assistant Professor of Medicine, UT Southwestern Medical Center (Dallas, TX), presents a diagnostic algorithmic approach for the establishment of a firm diagnosis of ATTR, with a focus on ATTR patients with heart manifestations (cardiomyopathy). Read More ›

Recognizing Clinical Clues of ATTR-CM
Clement C. Eiswirth, Jr., MD, FACC, Medical Director, Cardiomyopathy and Heart Failure Program, Ochsner Health, New Orleans, LA, examines clinical hints for consideration in the diagnosis of ATTR-CM. Read More ›

Evoking Suspicion of ATTR-CM: Warning Signs
Clement C. Eiswirth, Jr., MD, FACC, Medical Director, Cardiomyopathy and Heart Failure Program, Ochsner Health, New Orleans, LA, offers warning signs of ATTR-CM to consider for patients with heart failure. Read More ›

Setting Realistic Treatment Goals and Expectations for Patients with Transthyretin Amyloidosis
Ola Akinboboye, MD, MPH, MBA, Clinical Professor, Cardiology, Donald and Barbara Zucker School of Medicine at Hofstra University/Northwell Health and Medical Director, Laurelton Heart Specialists PC (Rosedale, NY) and Clement C. Eiswirth, Jr., MD, FACC, Medical Director, Cardiomyopathy and Heart Failure Program, Ochsner Health, New Orleans, LA, highlight the importance of early diagnosis and realistic expectations for patients being treated for transthyretin amyloidosis. Read More ›

Serum Transthyretin in the Management of TTR Amyloidosis
Justin L. Grodin, MD, MPH, Assistant Professor of Medicine, UT Southwestern Medical Center, Dallas, TX, explains the current and potentially future utilities of serum transthyretin (sTTR) as a biomarker in the management of transthyretin amyloidosis. Read More ›

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