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CME: Transthyretin amyloidosis (ATTR): Prompt diagnosis, contemporary management strategies, and their impact on patient outcomes


Activity Description / Statement of Need:

In this online, self-learning activity:

Transthyretin amyloidosis (ATTR) is a progressive, multisystem, life-threatening disorder characterized by the extracellular deposition of misfolded, insoluble amyloid fibrils. The role of the TTR protein is to transport thyroxine and retinol-binding proteins, and it is vital for cognition, nerve regeneration, and axonal growth. TTR itself is innately amyloidogenic even without the presence of genetic mutations, which may account for wild-type ATTR (wtATTR), while a hereditary form of ATTR (hATTR) may be passed to offspring through autosomal dominant inheritance. Left untreated, the average life expectancy of ATTR is 3 to 15 years from symptom onset.

This accredited educational activity would identify the critical components of the management process and offer solutions to close gaps in diagnosis and care, with the ultimate goals being the improvement of ATTR management, treatment adherence when applicable, and health and cost outcomes. 

Target Audience:

The following HCPs: neurologists, cardiologists, and hematologists; physician assistants, nurse practitioners, and pharmacists in the aforementioned areas of specialty; and any other HCPs with an interest in or who may clinically encounter patients with ATTR.

Commercial Support Disclosure: This program is supported by educational grants from Alnylam.

This activity is free of charge.

Release Date: December 29, 2021 -- Expiration Date: December 29, 2023

Faculty: Kourosh Rezania, MD


Faculty introduction, disclosures

ATTR, recognition, awareness, and missed opportunities in diagnosis

· Epidemiology of ATTR

· Genetics and subtypes of ATTR: hATTR & wtATTR

  • Pathophysiology

· ATTR symptomology and diagnosis

· Organ involvement and complications: cardiologic, neurologic, musculoskeletal

· Early symptoms

· Disease burden and impact on the patient

· Differential diagnosis & diagnostic tests

  • The risk of misdiagnosis and the impact of timely recognition

Treatment in patients with ATTR

  • Pharmacotherapy for ATTR and complications: medications and literature support
    • Polyneuropathy treatment with TTR synthesis inhibition: antisense oligonucleotids, small interfering RNA
    • Cardiomyopathy treatment: tetramer stabilization
  • Symptomatic management
  • Monitoring response to therapy and complications
  • Therapies in development
  • The need for a multidisciplinary approach to management, given the disease’s effects on multiple organ systems
  • Genetic counseling and family considerations
  • Patient cases

Summary, conclusions, and best practice recap

Learning Objectives

By the end of the session the participant will be able to:

  • Describe challenges in diagnosing ATTR early and the consequences of living with undiagnosed or misdiagnosed ATTR.
  • Recall methods of diagnosing ATTR and monitoring its complications.
  • Identify the presently available and emerging therapies for ATTR and its complications.
  • Formulate a treatment plan for a patient with ATTR and related complications and monitoring response to therapy.


ACCME Activity #201809242


Faculty Disclosure and Resolution of COI

As a provider of continuing medical education, it is the policy of ScientiaCME to ensure balance, independence, objectivity, and scientific rigor in all of its educational activities. In accordance with this policy, faculty and educational planners must disclose any significant relationships with commercial interests whose products or devices may be mentioned in faculty presentations, and any relationships with the commercial supporter of the activity. The intent of this disclosure is to provide the intended audience with information on which they can make their own judgments. Additionally, in the event a conflict of interest (COI) does exist, it is the policy of ScientiaCME to ensure that the COI is resolved in order to ensure the integrity of the CME activity. For this CME activity, any COI has been resolved thru content review ScientiaCME.

Faculty Disclosure:

Kourosh Rezania, MD, has received financial compensation as a consultant and/or being on the speaker’s bureau for Alnylam, Akcea, Grifols and Kabafusion.

Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, has no relevant financial disclosures.

Commercial Support Disclosure: This program is supported by educational grants from Alnylam.

All relevant financial relationships have been mitigated.

ScientiaCME adheres to the ACCME’s Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CE activity, including faculty, planners, reviewers or others are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity.

Faculty will not discuss off-label uses of a commercial product.


  • Read the learning objectives above
  • Take the Pre-Test (optional). Completion of the pre-test will help us evaluate the knowledge gained by participating in this CME activity.
  • View the online activity. You may view this is in more than one session, and may pause or repeat any portion of the presentation if you need to.
  • Minimum participation threshold: Take the post-test. A score of 70% or higher is required to pass and proceed to the activity evaluation.
  • Complete the activity evaluation and CME registration. A CE certificate will be emailed to you immediately.

Cultural/Linguistic Competence & Health Disparities

System Requirements

Windows 7 or above
Internet Explorer 8
*Adobe Acrobat Reader
Mac OS 10.2.8
Safari or Chrome or Firefox
*Adobe Acrobat Reader
Internet Explorer is not supported on the Macintosh

*Required to view Printable PDF Version

Perform Pre-Test (optional)

Please take a few minutes to participate in the optional pre-test. It will help us measure the knowledge gained by participating in this activity.

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Chronic inflammatory demyelinating polyneuropathy (CIDP): Optimizing pharmacotherapeutic management strategies