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The Latest Update on Metachromatic Leukodystrophy: Screening, Diagnosis, and Emerging Treatments to Improve Quality of Life of Patients

Premiere Date: Tuesday, October 18, 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:
Wednesday, October 18, 2023
Note: Credit Is No Longer Available

Faculty

Florian S. Eichler, MD (Moderator) Director, Center for Rare Neurological Disease Associate Professor of Neurology Massachusetts General Hospital Harvard Medical School Boston, MA

Laura A. Adang, MD, PhD  Assistant Professor of Child Neurology Children's Hospital of Philadelphia Philadelphia, PA

Rachel E. Hickey, MS, LCGC  (she/her/hers) Genetic Counselor, Care Coordinator of Leukodystrophy Clinic Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, IL

Statement of Need

Current and emerging therapies and treatment approaches can offer symptom relief and improved quality of life for patients with metachromatic leukodystrophy (MLD). But given MLD's rarity and health care professionals' lack of familiarity with the disease and its treatment, diagnosis is often inaccurate or delayed, negatively impacting outcomes.

During this 90-minute enduring material, expert panelists will discuss biomarkers that identify patients at the pre-symptomatic phase of the disease and share knowledge about caring for patients with MLD.

Learning Objectives

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

• Review the impact of diagnostic delay and misdiagnosis on patients with MLD.
• Indicate strategies for identifying patients with MLD at the pre-symptomatic phase of disease.
• Implement early screening for patients with suspected MLD based on symptoms and clinical presentations.
• Evaluate efficacy and safety of emerging therapies for MLD.

Financial Support

Supported by an educational grant from Takeda Pharmaceuticals U.S.A., Inc.

Target Audience

Physicians, PAs, nurse practitioners, nurses and pharmacists specializing in genetics, metabolism, neurology, pediatrics, internal medicine and/or primary care.

Credit Information

ABIM MOC Credit:
Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.5 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.

Learning Formats
Enduring material

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.

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 mitigated 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. Eichler reports the following financial relationships:

Advisory Board: Bluebird Bio, Inc. and SwanBio Therapeutics, Inc.

Consultant: Autobahn Therapeutics; Poxel S.A.; SwanBio Therapeutics, Inc.; Takeda Pharmaceuticals U.S.A., Inc.; Taysha Gene Therapies; and UpToDate, Inc.

Grants: European Leukodystrophy Association; National Institute of Neurological Disorders and Stroke (NINDS); and Undiagnosed Disease Network (UDN)

Research Support: Aspa Therapeutics; Bluebird Bio, Inc.; and Sio Gene Therapies

Speakers Bureau: American Society for Gene and Cell Therapy (ASGCT) and PRIME Education, LLC

Stock Shareholder (ownership interest): SwanBio Therapeuitics, Inc.

Other Financial or Material Support: UpToDate, Inc. Authorship Fees

Dr. Adang reports the following financial relationships:

Consultant: MEGMA; Orchard Therapeutics; and Takeda Pharmaceuticals U.S.A., Inc.

Grants: MSD Action Foundation; National Center for Advancing Translational Sciences (NCATS); National Institute of Neurological Disorders and Stroke (NINDS); and The Family Foundation

Ms. Hickey reports no financial relationships to disclose.

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

Jeffrey Helfand, DO (peer reviewer)

Thomas Mitchell (planning committee)

Kellie Busby, PharmD (planning committee)

Susan H. Yarbrough, CHCP (planning committee)

Sandra Caballero, PharmD (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).