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Emerging Gene Therapies in the Fight Against ALS

Premiere Date: Thursday, March 24, 2022

This activity offers CE credit for:

  1. ABIM (MOC)
  2. Medicine (accme)
  3. Nursing (ANCC)
  4. Pharmacy (acpe)
  5. PA (aapa)
  6. Other

All other clinicians will receive a Certificate of Attendance stating this activity was certified for AMA PRA Category 1 Credit™

Credit Expiration Date:
Friday, March 24, 2023
Note: Credit Is No Longer Available


Jinsy A. Andrews, MD, MSc, FAANJinsy A. Andrews, MD, MSc, FAAN (Moderator)
Director of Neuromuscular Clinical Trials, Department of Neurology
Columbia University Irving Medical Center
New York, NY

Jeremy M. Shefner, MD, PhDJeremy M. Shefner, MD, PhD 
Professor and Chair of Neurology
Barrow Neurological Institute
Phoenix, AZ

Statement of Need

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease with no known cure. The estimated 16,000 – 20,000 people in the United States living with ALS experience advancing weakness leading to paralysis of the muscles required to move, speak, and breathe. A lack of effective intervention in this fatal disease has motivated an explosion of recent discoveries in the genetic etiology and pathophysiology of ALS. However, clinicians may not be familiar with the latest data on emerging therapies utilizing newly discovered genetic targets.

Given the modest efficacy of current ALS treatments, access to emerging therapies is critical. Clinicians must be up-to-date on efficacy, safety, clinical trial results, and access pathways for their patients. Join Dr. Andrews and Dr. Shefner in part 3 of this CMEOutfitter’s snack series as they discuss best practices for navigating the clinical trial landscape of ALS gene therapies.

Learning Objectives

At the end of this CE activity, participants should be able to:

  • Assess safety and efficacy of emerging agents.

Financial Support

Supported by an educational grant from Biogen MA, Inc.

Target Audience

Adult neurologists, geneticists, genetic counselors, primary care physicians, PAs, nurse practitioners, nurses, physical therapists, occupational therapists, otolaryngologists

Credit Information

Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 0.75 medical knowledge MOC points in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC points equivalent to the amount of CME credits claimed for the activity. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit.

Royal College MOC:
Through an agreement between the Accreditation Council for Continuing Medical Education and the Royal College of Physicians and Surgeons of Canada, medical practitioners participating in the Royal College MOC Program may record completion of accredited activities registered under the ACCME’s “CME in Support of MOC” program in Section 3 of the Royal College’s MOC Program.

MIPS Improvement Activity:
This activity counts towards MIPS Improvement Activity requirements under the Medicare Access and CHIP Reauthorization Act of 2015 (MACRA). Clinicians should submit their improvement activities by attestation via the CMS Quality Payment Program website.

Disclosure Declaration

It is the policy of CME Outfitters, LLC, to ensure independence, balance, objectivity, and scientific rigor and integrity in all of their CE activities. Faculty must disclose to the participants any relationships with commercial companies whose products or devices may be mentioned in faculty presentations, or with the commercial supporter of this CE activity. CME Outfitters, LLC, has evaluated, identified, and attempted to resolve any potential conflicts of interest through a rigorous content validation procedure, use of evidence-based data/research, and a multidisciplinary peer review process. The following information is for participant information only. It is not assumed that these relationships will have a negative impact on the presentations.

Dr. Andrews reports the following financial relationships:

Consultant: AL-S Pharma AG; Amylyx Pharmaceuticals Inc.; Biogen Idec; Cytokinetics; Denali Therapeutics; Neurosense Therapeutics; Orphazyme; and Wave Life Sciences

Research Support: Alexion Pharmaceuticals, Inc.;AZTherapies Inc.; Biogen Idec; Biohaven Pharmaceuticals; Clene Nanomedicine, Inc.; Cytokinetics; Healey Center at Massachusetts General Hospital; National Institutes of Health; Novartis AG; Orion; and Ra Pharmaceuticals, Inc.

Dr. Shefner reports the following financial relationships:

Consultant: Amylyx; Apic Bio; NeuroSense Therapeutics; Cytokinetics Incorporated; Denali Therapeutics; EMD Serono; GlaxoSmithKline; Helixmith; Mitsubishi Tanabe Pharma America; Novartis; Orphazyme; Orthogonal Therapeutics; Pinteon; RRD; Sanofi; and SwanBio Therapeutics, Inc.

Grants: National Institutes of Health (NIH); ALS Association; and Mitsubishi Tanabe Pharma America

Research Support: Alector, Inc.; Alexion Pharmaceuticals, Inc.; Amylyx; Biogen; Biotie Therapies (now Acorda Therapeutics); Cytokinetics Incorporated, Inc.; Ionis Pharmaceuticals; MediciNova, Inc.; Mitsubishi Tanabe Pharma America; and Orphazyme

The following peer reviewer and CME Outfitters staff have no financial relationships to disclose:

  • Jeffrey Helfand, DO (peer reviewer)
  • Kellie Busby, PharmD (planning committee)
  • Evan Luberger (planning committee)
  • Susan H. Yarbrough, CHCP (planning committee)
  • Sharon Tordoff (planning committee)

  • Faculty of this CE activity may include discussions of products or devices that are not currently labeled for use by the FDA. The faculty have been informed of their responsibility to disclose to the audience if they will be discussing off-label or investigational uses (any uses not approved by the FDA) of products or devices.

    Questions about this activity? Call us at 877.CME.PROS (877.263.7767).


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