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CME: Hereditary transthyretin amyloidosis (ATTR) treatment strategies: best practices and emerging therapies


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. As TTR is a protein present in human serum, and its role is to transport thyroxine and retinol-binding proteins, it is vital for behavior, cognition, nerve regeneration, and axonal growth. TTR itself is innately amyloidogenic even without the presence of genetic mutations. The familial variant of the disease (hATTR) is ATTR that is passed to offspring through autosomal dominant inheritance, whereas the wild type variant (wtATTR) refers to ATTR that occurs independently of genetic mutations. hATTR may present as late as mid-adulthood, but its symptoms usually start between the ages of 2 and 10 years. Left untreated, the average life expectancy of ATTR is 3 to 15 years from symptom onset.



Target Audience:

The following HCPs: neurologists, cardiologists, and hematologists; physician assistants, nurse practitioners, nurses, 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 an educational grant from Akcea Therapeutics.

This activity is free of charge.

Release Date: July 02, 2021 -- Expiration Date: July 01, 2023

Faculty: Michelle Kaku, MD -- Deepa Gopal, MD, MS


Introduction content: Cursory refresher and review of hATRR, including contrast wtATTR

  • Epidemiology and etiology of hATRR
    • Statistics
    • Age
    • Genetics
    • Symptomology and diagnosis of TRR amyloidosis
      • Pathophysiology
      • Onset and classification
      • Signs and symptoms
      • Differential diagnoses
      • Diagnostic tests
      • Progression of disease
      • Complications

Treatment in patients with TRR amyloidosis

  • Pharmacotherapy: patisiran, symptomatic management
  • Drugs in development
  • Emerging strategies and trends in recent years
  • Best practice: putting it all together
  • Patient case(s)

Learning Objectives

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

  • Describe the pathophysiology of ATTR such that it might inform treatment mechanisms
  • Describe available therapies used for treatment of ATTR and explain current literature supporting use of those therapies
  • Design and implement an appropriate therapeutic plan for treatment of ATTR
  • Describe future therapies currently being investigated for the treatment of ATTR


ACCME Activity #202657536


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: Michelle Kaku, MD, Assistant Professor of Neurology, Boston University, has no relevant financial disclosures.

Deepa Gopal, MD, MS, Assistant Professor of Cardiovascular Medicine, Boston University, has no relevant financial disclosures.

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

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.


Commercial Support Disclosure:  This program is supported by an educational grant from Akcea Therapeutics


  • 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.