ATTR Wild Type and Hereditary Amyloidosis

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Amyloidosis Support Groups 8th ATTR/Hereditary Amyloidosis Meeting
October 24-26, 2025

Attendees Thus Far:
Kevin Michael Alexander MD, Stanford
John Berk MD, BU
Thomas Brannagan MD, Columbia
Emily Brown, JH
Monica Fawzy Bryant Esq, Triage
John Clarke, Stanford
Noel Dasgupta MD, IU
Sharmila Dorbala MD, Harvard
Morie Gertz MD, Mayo
Justin Grodin MD, UTSW
Martha Grogan MD, Mayo
Mazen Hanna MD, CC
Jackie Jacobi MD, Mayo
Dan Judge MD, MUSC
Chafic Karam MD, UPenn
Nelson Leung MD, Mayo
Melissa Lyle MD, Mayo
Ahmad Masri MD, OHSU
Emily Martin PhD, Univ. of Tenn.
Jose Nativi MD, Mayo
Rick Ruberg MD, BU
Nitasha Sarswat MD, Univ. of Chicago
Farooq Sheikh MD, Medstar
Barry Trachtenberg MD, Houston Methodist
Jonathan Wall PhD, Univ. of Tenn.
David Wolinski MD, CC

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PAST EVENTS ON VIDEO

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ASG ATTR Webinar
March 9, 2024

Recording of the Amyloidosis Amyloidosis Support Groups webinar on ATTR amyloidosis recorded on March 9, 2024.

MODERATOR:

Muriel Finkel, President, Amyloidosis Support Groups

Speakers:

Tony Urey, MD, Cardiologist, Assistant Professor of Medicine, UC San Diego School of Medicine, La Jolla, CA
Chafic Karam, MD, Neurologist, Associate Professor of Neurology, University of Pennsylvania, Philadelphia, PA
Jonathan Wall, PhD, Professor, University of Tennessee Graduate School of Medicine, Knoxville, TN
Paula Schmitt, Executive Director, Amyloidosis Support Groups
*Gabriela Tamariz, MD, Medical Science Liaison, Alnylam Pharmaceuticals, Cambridge, MA
*Adam Castano, MD, MS, Vice President, Global Clinical Development, ATTR Amyloidosis, BridgeBio Pharma, New York, NY
*Hannah Pope, PharmD, MHA, Sr. Medical Science Liaison, BridgeBio Pharma, St. Louis, MO
*Liron Walsh, MD, Head of Development, In Vivo Programs, Intellia Therapeutics, Inc., Cambridge, MA
*Sidsel Hoier Gamborg Moller, MD, PhD, Global Medical Manager, Novo Nordisk, Copenhagen, Denmark
*Alyssa A. Hawranko, PhD, Executive Medical Science Liaison, ATTR – Amyloidosis, US Medical Affairs, AstraZeneca, LP
*Cristina Quarta, MD, PhD, Senior Medical Director, Alexion, AstraZeneca Rare Disease, Boston, MA
*Lori Baylor, PhD, Medical Director – Rare Cardiology, US Medical Affairs, Rare Disease, Pfizer Inc., Boston, MA

* Please note that the presentations from pharmaceutical representatives, plus certain Q&A participation, have been excluded from this recording because they were only authorized for use during the live webinar and have not been authorized for post-webinar publication and distribution

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Chapters:
0:00 Welcome and Speaker Introductions
3:09 ATTR Treatment Chart
3:30 Dr. Tony Urey, Current FDA Approved Treatments for ATTR Amyloidosis
21:31 Dr. Chafic Karam, hATTR Polyneuropathy Treatments
42:15 Dr. Tony Urey, Drugs in Clinical Trials (silencers)
59:00 Q&A with Drs. Urey, Karam
1:17:46 Paula Schmitt, Financial Assistance Update
1:26:11 Dr. Tony Urey, Investigational and Anti-fibril Therapies
1:38:17 Q&A 2 with Drs. Urey, Karam
1:44:47 Dr. Jon Wall, Diagnosing Cardiac Amyloid using 99mTc-p5+14 SPECT/CT Imaging
1:59:50 Muriel Finkel & Dr. Urey on BridgeBio ACT-Early trial, use of Tafamidis in BridgeBio ATTRibute-CM study
2:01:44 Muriel Finkel questioning whether pacemakers exclude participants from enrolling in ATTR trials (answer: they usually don't)

This webinar video has been edited for content and time.

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Cardiac Amyloidosis Webinar
January 20, 2024

Recording of the Amyloidosis Support Groups webinar on Cardiac Amyloidosis in AL (light chain) and ATTR (transthyretin) Amyloidosis from January 20, 2024. This webinar recording has been edited for content and time.

MODERATOR:

Muriel Finkel, President, Amyloidosis Support Groups

SPEAKERS:

Melissa Lyle, MD, FACC, Assistant Professor of Medicine, Cardiology Lead, Multidisciplinary Amyloid Clinic, Mayo Clinic, Jacksonville, FL

Justin Grodin, MD, MPH, FACC, Associate Professor of Medicine, Medical Director, Internal Medicine Clinical Trials Unit, University of Texas Southwestern Medical Center, Dallas, Texas

Barry Trachtenberg, MD, FACC, Advanced Heart Failure Cardiology, Houston Cardiovascular Associates, J.C. Walter Transplant Center at Houston Methodist Hospital, Houston, TX

Paula Schmitt, Executive Director, Amyloidosis Support Groups

*Gustavo Buchele, MD, PhD, Senior Director, Clinical Development, Alexion Pharmaceuticals, Boston, MA

*Mark McGovern, RN, CCRN, Vice President Clinical Operations, Eidos Therapeutics, a subsidiary of BridgeBio Pharma, San Francisco, CA

*Joy Olbertz, PharmD, PhD, Senior Director, Clinical Scientist, Intellia Therapeutics, Inc., Cambridge, MA

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Chapters
0:00 Welcome
2:53 Speaker Introductions
3:52 ATTR and AL Treatment Charts
4:34 Melissa Lyle: Cardiac Amyloidosis: Heart Failure Symptoms and Treatment
32:23 Justin Grodin: Arrhythmias, Pacemakers, & Defibrillators in Cardiac Amyloidosis
1:00:05 Barry Trachtenberg: Advanced Heart Failure Therapies in Cardiac Amyloidosis
1:32:27 Panel Discussion/Q&A
1:57:54 Paula Schmitt: Financial Assistance
2:07:39 Panel Discussion/Q&A cont'd
2:11:13 Pharma Trial Information
2:11:43 Panel Comments on Pharmaceutical Trials

*Live webinar only. Presentations and commentary from these speakers were authorized for the live webinar only and are excluded from this video recording.

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Amyloidosis Support Groups 7th ATTR/Hereditary Amyloidosis Meeting - October 27-29, 2023

These are the videos of the presenting physicians, researchers, and advocates from ASG's biennial ATTR and Hereditary amyloidosis patient meeting from Friday October 27 through Sunday October 29. All presentations took place on Saturday, October 28, 2023.

WATCH ON YOUTUBE: Video Playlist from ASG ATTR/Hereditary 2023 Meeting for Patients and Caregivers

Links to each presentation

ATTR: Molecules That Cause and Molecules That Treat TTR Amyloidosis
Avi Chakrabartty, PhD
Professor Emeritus
Department of Medical Biophysics
University of Toronto, Toronto, Canada

ATTR Cardiac Staging: Biomarkers and Imaging
Martha Grogan, MD
Director, Cardiac Amyloidosis Clinic
Mayo Clinic, Rochester, MN

ATTR Cardiac Amyloidosis: Treating the Symptoms
Mat Maurer, MD
Arnold and Arlene Goldstein Professor of Cardiology
Professor of Medicine
Columbia University Irving Medical Center, New York, NY

Imaging in ATTR Cardiac Amyloidosis: How it is used for Diagnosis and Treatment Response
Frederick L. Ruberg, MD
Associate Chief for Cardiovascular Medicine
Boston University Medical Center
Professor of Medicine
Boston University School of Medicine, Boston, MA

The Future of Imaging
Jonathan Wall, PhD
Director, Amyloidosis and Cancer Theranostics Program
University of Tennessee Graduate School of Medicine, Knoxville, TN

ATTR Peripheral Neuropathy Treatments
Chafic Karam, MD
Associate Professor, Clinical Neurology
University of Pennsylvania, Philadelphia, PA

ATTR Peripheral Neuropathy Symptoms
Thomas Brannagan, MD
Director, Peripheral Neuropathy Center Neurological Institute, and
Professor of Neurology
Columbia University, Vagelos College of Physicians and Surgeons
New York, NY

ATTR: Alternative Treatments and When to Start
Morrie Gertz, MD, presenting on behalf of John Berk, MD
Morrie Gertz, MD
Roland Seidler Jr. Professor, College of Medicine, and
Mayo Distinguished Clinician,
Mayo Clinic, Rochester, MN

John Berk, MD
Assistant Director, Amyloidosis Center
Boston University Medical Center, and
Professor of Medicine
Boston University School of Medicine, Boston, MA

ATTR: What Treatments are in our Future
Ahmad Masri, MD, MS
Director, Cardiac Amyloidosis Program
Director, Hypertrophic Cardiomyopathy Center
Assistant Professor of Medicine
Oregon Health & Science University, Portland, OR

ATTR Amyloidosis & the Gut: GI Symptoms & Treatments
John O. Clarke, M.D.
Director, Esophageal Program
Clinical Professor of Medicine
Division of Gastroenterology and Hepatology
Stanford University, Stanford, CA

ATTR: Genetics of Hereditary Transthyretin Amyloidosis
Emily Brown, MGC, CGC
Certified Genetic Counselor
Center for Inherited Heart Disease
Johns Hopkins University, Baltimore, MD

The Burden of ATTR Amyloidosis on Patients and Their Caregivers: ARC Survey Results
Kristen Hsu
Executive Director of Research
Amyloidosis Research Consortium, Newton, MA
https://arci.org

Amyloidosis Speakers Bureau (ASB)
Deb Boedicker
Board Member, MacKenzie's Mission
Operating Committee Member, Amyloidosis Speakers Bureau

Amyloidosis Patient Registry
Robert Gibson
Director, Special Projects
Amyloidosis Support Groups

Insurance and Financial Assistance
Monica Fawzy Bryant, ESQ
Health Care Rights Attorney
Co-founder and Chief Operating Officer, Triage Cancer, Chicago, IL

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Explanation of Wild Type ATTR

hATTR amyloidosis is a rare condition that occurs when an abnormal protein called amyloid builds up in tissue or organs like the heart, nervous system, and digestive tract. Watch our past online webinars on ATTR listed below. They are essentially a virtual patient education program that you can view on demand today (or at your convenience) and allows you to hear from doctors, advocates and people living with hATTR amyloidosis to learn about:

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Amyloidosis Support Groups ATTR Amyloidosis Webinar - January 21, 2023

Moderater:
Muriel Finkel, President, Amyloidosis Support Groups

Participants:
Mathew S. Maurer, MD, Cardiologist, Director, Cardiac Amyloidosis Center, Professor of Medicine, Columbia University Medical Center
Chafic Karam, MD, Neurologist, Associate Professor of Neurology, University of Pennsylvania
*Joy Olbertz, PharmD, PhD, Senior Director, Clinical Scientist, Intellia Therapeutics
*Anne Ersbøll MD, PhD, Senior Global Medical Manager, Global Medical Affairs, Cardiovascular Diseases, Novo Nordisk
*Gustavo Buchele, MD, PhD, Executive Director, Clinical Development, Ionis Pharmaceuticals
Craig Spolsky, Advocacy & Professional Relations Director, Pfizer, Inc.
*Steven Roblin, PhD, US Medical Director, Alnylam Pharmaceuticals
Jocelyn Ashford, Vice President, Patient Advocacy, BridgeBio Pharma
Paula Schmitt, Executive Director, Amyloidosis Support Groups

*Presentations and discussions by these participants were only authorized for use during the live webinar, not for publication on YouTube. Their contributions have been removed from this video.

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Chapters
0:00 Welcome and Introduction of Participants
3:24 ATTR Treatment Chart
3:52 Dr. Mat Maurer, Systemic Amyloidosis
27:14 Dr. Chafic Karam, Neurological Complications of ATTR Amyloidosis
56:55 Q&A Session One
1:36:24 Muriel Finkel, Info on the 2023 ASG ATTR Patient Conference this October
1:38:17 Paula Schmitt, Patient Support and Financial Assistance
1:48:25 Jocelyn Ashford, BridgeBio Update
1:50:44 Craig Spolsky, Update on Pfizer Patient Affairs Liaisons for ATTR
1:56:36 Q&A Session Two

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ASG ATTR Webinar, July 23, 2022

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ASG ATTR Webinar, March 19, 2022

Moderator: Muriel Finkel, President, Amyloidosis Support Groups

Presenters and Panelists

Paula Schmitt, Executive Director, Amyloidosis Support Groups
Mathew S. Maurer, MD, Cardiologist, Director, Cardiac Amyloidosis Center, Professor of Medicine, Columbia University Medical Center
Nitasha Sarswat, MD, Director of Infiltrative Cardiomyopathy Program, University of Chicago Medicine
Lynn Punnoose, MD, Director, Vanderbilt Advanced Heart Failure/Transplant Fellowship Program, Vanderbilt University Medical Center
Sami Khella, MD, Chief of Neurology, Penn Presbyterian Medical Center
Michael Maitland, MD, PhD, Sr. Director, Clinical Development, Intellia Therapeutics
*Colleen Moffitt, PharmD, MS, US ATTR Medical Director, Alnylam Pharmaceuticals
Gustavo Buchele, MD, PhD, Executive Director, Clinical Development, Ionis Pharmaceuticals
*Abbas Ebrahim, MD, Sr. Medical Director, US Medical Affairs, Rare Disease, Pfizer
*Lori Baylor, PhD, Medical Director, Rare Cardiology, US Medical Affairs, Rare Disease, Pfizer
Spencer Guthrie, Cofounder & Chief Operating Officer, Attralus, Inc., South San Francisco, CA

*Presentations made by Alnylam and Pfizer during the webinar are not yet authorized for publication on YouTube. When and if authorized, ASG will make those presentations available on the YouTube channel.

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Northeast ATTR Webinar, July 10, 2021

Presentations

Welcome and Panelist Introductions, Muriel Finkel, ASG President
Transthyretin Cardiac Amyloidosis (ATTR-CM) Signs, Symptoms, and Diagnostic Approach, Farooq Sheikh, MD, FACC
Treatment for ATTR Cardiac Amyloid, John Berk, MD
Symptom Mangement in Cardiac Amyloidosis: Back to Basics, Frederick L. Ruberg, MD
Alnylam Clinical Trial Update, Gabriela Tamariz, MD
Eidos Clinical Trial Update, Makenna Paule
Ionis Clinical Trial Update, Gustavo Buchele, MD, PhD
Question and Answer Session 1, Panelists
Peripheral Neuropathy Symptoms, Diagnostic Tests, and Approved Treatments, Thomas Brannagan, MD
Symptom Management in Amyloid Neuropathy, Chafic Karam, MD
Alnylam Update on Clinical Trial for Hereditary TTR, Gabriela Tamariz, MD
Ionis Update on Clinical Trial for Hereditary TTR, Gustavo Buchele, MD, PhD
Intellia CRISPR CAS9 Update, Jeff Cehelsky, BS Pharm, MBA
Question and Answer Session 2, Panelists
Financial Assistance, Paula Schmitt, ASG Executive Director

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Pacific Northwest ATTR Virtual Support Group Webinar, March 13, 2021

Presentations

Introduction and Speaker Bios, Muriel Finkel, President, Amyloidosis Support Groups
Agenda, Dr. Ahmad Masri
ATTR in the Heart, Dr. Deirdre Mooney
Treatment for ATTR Cardiomyopathy, Dr. Richard Cheng
Amyloid Transthyretin (ATTR) Amyloidosis in the Nerves, Dr. Jane Distad
Amyloidosis — Why Did I Have to Get All of Those Tests? Dr. Edward Libby
ATTR Treatment: Navigating Through the Complexity of the Healthcare System, Dr. Hongya Chen
What do All These Neurological Tests Mean? Dr. Orly Moshe-Lilie
Transthyretin Amyloidosis: Advances and New Therapies, Dr. Ahmad Masri
Question and Answer Session, Panelists
Wrap Up and Final Thoughts, Panelists

Panelists

Muriel Finkel, ASG President
Ahmad Masri, MD, MS, Cardiology, Director of the Hypertrophic Cardiomyopathy Center at Oregon Health & Science University, Portland, OR
Deirdre Mooney, MD, MPH, FACC, Cardiologist at Sacred Heart Medical Center, Providence Health & Services, Spokane, WA
Richard Cheng, MD, MSc, Cardiologist/Cardio-oncologist, Assistant Professor of Cardiology, University of Washington, Seattle, WA
B. Jane Distad, MD, Director, EMG and Associate Professor, Department of Neurology, University of Washington, Seattle, WA
Orly Moshe-Lilie, MD, Neurologist, Neuromuscular Fellow at Oregon Health & Science University, Portland, OR
Edward N. Libby, MD, Hematology/Oncology, Associate Professor, Medical Oncology, University of Washington, Seattle, WA
Hongya Chen, PharmD, Cardiology, Oregon Health & Science University, Portland, OR

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ATTR Treatments Amyloidosis Support Group Webinar, December 5, 2020

Presentations*

Introduction, Agenda plus Overview of TTR Amyloidosis - Muriel Finkel, President, ASG and Brett Sperry, MD
Ionis' Tegsedi Update plus Q&A on TTR Silencers - Gustavo Buchele, MD and Panelists
Pfizer's Vyndaqel and Vyndamax (tafamidis) - Adam Castano, MD
Corino's CRX-1008 - Michael Roberts, PhD
Q&A on TTR Stabilizers and Silencers - Panelists
ASG Update: Financial Assistance, oneAMYLOIDOSISvoice, Patient Registry - Paula Schmitt, ASG Executive Director
Intellia's CRISPR ATTR Treatment - Jeffrey Cehelsky, VP of Development Operations
ATTR Amyloid Imaging - Jonathan Wall, PhD
General Q&A Session 1 - Panelists
Clinical Trials Update* - Pfizer, Corino, Ionis, Intellia
General Q&A Session 2 - Panelists

*The webinar presentations and clinical trial updates by Alnylam about Onpattro (patisiran) and by Eidos about Acoramidis (AG10) are unavailable as they are not authorized for post-webinar distribution.

Speakers and Panelists

Brett Sperry, MD, St. Luke's Mid America Heart Institute and Assistant Professor of Medicine, University of Missouri, Kansas City, MO
Akshay Vaishnaw, MD, PhD, President, Research and Development, Alnylam Pharmaceuticals, Cambridge, MA
Steven Roblin, PhD, Medical Director, US TTR Lead, Alnylam Pharmaceuticals, Cambridge, MA
Gustavo Buchele, MD, PhD, Medical Director, Ionis Pharmaceutical, Inc., Carlsbad, CA
Adam Castano, MD, MS, Senior Medical Director, ATTR Cardiac Amyloidosis, Pfizer Inc., New York, NY
Alan Cohen, MD, Vice President, Global Medical Affairs, Eidos Therapeutics, Inc., San Francisco, CA
Michael Roberts, PhD, Chief Executive Officer, Corino Therapeutics, Inc., Charlotte, NC
Jeffrey Cehelsky, BS Pharm, MBA, Vice President of Development Operations, Intellia Therapeutics, Cambridge, MA
Jocelyn Ashford, Head of Global Patient Advocacy, Eidos Therapeutics, Inc., San Francisco, CA
Jonathan Wall, PhD, Professor, University of Tennessee Graduate School of Medicine, Knoxville, TN
Chafic Karam, MD, Associate Professor of Neurology, University of Pennsylvania, Philadelphia, PA
Nitasha Sarswat, MD, Director of Infiltrative Cardiomyopathy Program, University of Chicago, Chicago, IL

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hATTR and ATTRwt Amyloidosis Support Group Meeting, August 15, 2020

Aug 15 2020 Webinar Playlist

Presentations

Introduction, Memorial Tribute and Participant Bios - Muriel Finkel, President, ASG and Tracey Deutsch, International Liaison, ASG
Amyloidosis and the Heart - Martha Grogan, MD
Neurological Complications of ATTR Amyloidosis - Chafic Karam, MD
Amyloidosis and the Gut - John Clarke, MD
Health Insurance - Monica Bryant, Esq
Genetic Counseling for TTR Amyloidosis - Katie Agre, MS, LCGC
ASG Updates: Financial Assistance, oneAMYLOIDOSISvoice, Patient Registry- Paula Schmitt, ASG Executive Director and Bob Gibson, ASG Special Projects Director
Q&A Session - Speakers and Panelists

Speakers

Martha Grogan, MD, Director, Cardiac Amyloid Clinic, Mayo Clinic, Rochester, MN
Chafic Karam, MD, Professor of Neurology, Oregon Health & Science University
John Clarke, MD, Director, Esophageal Program, Clinical Associate Professor of Medicine, Stanford University
Monica Bryant, Esq., Health Care Rights Attorney, Triage Cancer
Katie Agre, MS, Licensed Certified Genetic Counselor, Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN

Panelists

Kevin Alexander, MD, Assistant Professor of Medicine, The Stanford University Medical Center
Vincent Lau, MD, Assistant Professor of Neurology, Boston University School of Medicine, Boston, MA
Celestial Miller, MDA,RDN,CSO,CNSC,CD, University of Utah Healthcare, Salt Lake City, UT
Jignesh K. Patel, MD, PhD, Cedars-Sinai California Heart Center, Beverly Hills, CA
Maria M. Picken, MD, PhD, Professor, Loyola University Medical Center, Chicago, IL
Nitasha Sarswat, MD, Director of Infiltrative Cardiomyopathy Program, University of Chicago
Ron Witteles, MD, Co-Director, Stanford Amyloid Center, The Stanford University Medical Center

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ATTR Amyloidosis Support Group Meeting, April 25, 2020

Apr 25 2020 ATTR Webinar Playlist

Presentations

ATTR Cardiac Amyloidosis David Wolinsky, MD, Director Cardiac Amyloid Clinic, Cleveland Clinic, Florida
Peripheral and Autonomic Neuropathy Chafic Karam, MD, Professor of Neurology, Oregon Health & Science University
Renal Amyloidosis Andrea Havasi, MD, Amyloidosis Center, Boston University School of Medicine
Imaging Systemic Amyloidosis (ATTR) Johnathan Wall, MD, University of Tennessee Graduate School of Medicine
Questions and Answers, Panelists