Accreditation/Credit Designation

Physicians’ Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Physicians’ Education Resource®, LLC, designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Physicians’ Education Resource®, LLC, is approved by the California Board of Registered Nursing, Provider #16669, for 1.0 Contact Hour.

Acknowledgment of Commercial Support

This activity is supported by an educational grant from Sanofi Genzyme.

Clinical Briefings™: Diagnosing and Treating MPS I: Opportunities to Improve Prognosis

Release Date: November 27, 2019
Expiration Date: November 27, 2020
Media: Internet - based

Activity Overview

This CME online activity, Clinical Briefings™: Diagnosing and Treating MPS I: Opportunities to Improve Prognosis, features written content complimented by audio commentary from Paul Orchard, MD, a pediatrician and expert in the field of cell therapies for inherited metabolic diseases. This activity provides an overview of lysosomal storage disorders with a focus on mucopolysaccharidosis type I (MPS I), characteristic features and diagnostic assessment in this setting, and current and novel treatments for individuals who have received a diagnosis of this disease.

Benefits of Participating

Upon completion of this educational activity, you will:

  • Recognize characteristic features of MPS I for early diagnosis
  • Determine newborn screening recommendations in the state where you practice
  • Explain care decisions to patients and caregivers
  • Discuss clinical data of new therapies for MPS I with your colleagues
  • Learn something new about lysosomal storage disorders and earn continuing medical education (CME) credit.

Acknowledgement of Commercial Support

This activity is supported by an educational grant from Sanofi Genzyme.

Instructions for This Activity and Receiving Credit

  • You will need to log in to participate in the activity.
  • Each presentation may contain an interactive question(s). You may move forward through the presentation; however, you may not go back to change answers or review video/content until you finish the presentation.
  • At the end of the activity, “Educational Content/Audio” will be available for your reference.
  • To receive a CME/CE Certificate, you must complete the activity.
  • Complete the Posttest and pass with a score of 70% or higher, complete the Evaluation, and then click on “Request for Credit.” You may immediately download a CME/CE Certificate upon completion of these steps.

Target Audience

This online educational activity is directed toward pediatricians and medical specialists who have the opportunity to recognize MPS 1 such as ophthalmologists, growth specialists, orthopedic surgeons, general surgeons, ear nose and throat specialists, cardiologists, pulmonologists, geneticists. Additionally, nurse practitioners (NPs), physician assistants (PAs), nurses and other health care professionals who would be involved in the diagnosis and management of MPS I will be invited to participate.

Learning Objectives

Upon successful completion of this educational activity, you should be better prepared to:

  • Review the pathophysiology of MPS I
  • Recognize the clinical presentation and diagnostic evaluation of MPS I
  • Evaluate the limitations in the historical standard of care
  • Discuss the efficacy and safety of enzyme replacement therapy and emerging therapies for patients with MPS I

Faculty, Staff, and Planners’ Disclosures

Faculty

Paul Orchard
Paul Orchard, MD
Medical Director of the Inherited Metabolic & Storage Disease Bone Marrow Transplantation Program Professor
Division of Pediatric Bone Marrow Transplantation
University of Minnesota
Minneapolis, MN

Disclosures: Grant Research Support: Magenta Therapeutics, Immusoft Corporation, bluebird bio.

The staff of Physicians' Education Resource®, LLC (PER®) have no relevant financial relationships with commercial interests to disclose.

Disclosure Policy and Resolution of Conflicts of Interest (COI)

As a sponsor accredited by the ACCME, PER® makes it a policy to ensure fair balance, independence, objectivity, and scientific rigor in all its CME/CE activities. In compliance with ACCME guidelines, PER® requires everyone who is in a position to control the content of a CME/CE activity to disclose all relevant financial relationships with commercial interests. The ACCME defines relevant financial relationships as financial relationships in any amount occurring within the past 12 months that create a COI.

Additionally, PER® is required by ACCME to resolve all COI. PER® has identified and resolved all COI prior to the start of this activity by using a multistep process.

Off-Label Disclosure and Disclaimer

This CME/CE activity may or may not discuss investigational, unapproved, or off-label use of drugs. Participants are advised to consult prescribing information for any products discussed. The information provided in this CME/CE activity is for continuing medical and nursing education purposes only and is not meant to substitute for the independent clinical judgment of a physician and nurse relative to diagnostic or treatment options for a specific patient’s medical condition.

The opinions expressed in the content are solely those of the individual faculty members and do not reflect those of PER® or the company that provided commercial support.

PER Pulse™ Recaps

1 of 3

Clinical Briefings™: Diagnosing and Treating Mucopolysaccharidosis Type I: Opportunities to Improve Prognosis is a continuing medical education–certified activity in which Paul Orchard, MD, a pediatrician and expert in the field of cell therapies for inherited metabolic diseases, discusses diagnosis and treatment options when caring for patients with mucopolysaccharidosis type I (MPS I).

This online activity begins with a brief overview of the pathophysiology of MPS I. Below are highlights of the topics covered during the activity.

  • Description of inherited lysosomal disorders
  • Effects of the enzyme deficiency characteristic of MPS I1-3
  • Past and current classifications of MPS I

References

  1. Neufeld EF, Muenzer J. The mucopolysaccharidoses. In: Valle D, Beaudet AL, Vogelstein B, Kinzler KW, Antonarakis SE, Ballabio A, Gibson K, Mitchell G, eds. The Online Metabolic and Molecular Bases of Inherited Disease. New York, NY: McGraw-Hill; 2014. https://ommbid.mhmedical.com/content.aspx?bookid=971§ionid=62642135. Accessed October 30, 2019.
  2. Mucopolysaccharidosis type I. US National Library of Medicine website. https://ghr.nlm.nih.gov/condition/mucopolysaccharidosis-type-i. Reviewed December 2012. Published November 12, 2019. Accessed November 13, 2019.
  3. Clarke LA. Mucopolysaccharidosis type I. National Organization for Rare Disorders website. https://rarediseases.org/rare-diseases/mucopolysaccharidosis-type-i/. Published 1986. Updated 2019. Accessed October 31, 2019.

2 of 3

Clinical Briefings™: Diagnosing and Treating Mucopolysaccharidosis Type I: Opportunities to Improve Prognosis is a continuing medical education–certified activity in which Paul Orchard, MD, a pediatrician and expert in the field of cell therapies for inherited metabolic diseases, discusses diagnosis and treatment options when caring for patients with mucopolysaccharidosis type I (MPS I).

Below are some of the discussion points featured in this online activity:

  • Difference between severe and attenuated forms of MPS I1,2
  • Common and distinguishing features of the 2 forms of MPS I2
  • Specific diagnostic tests used for definitive diagnoses of MPS I2
  • Nuances in newborn screening3

“In 2016, the Recommended Uniform Screening Panel [RUSP] recommended approval of MPS I as a disease that may be screened for. It is important to recognize, however, that each state makes its own determinations on what they are going to screen for, and so the approval of [MPS I] on the RUSP is not binding to the state.”
—Paul Orchard, MD

References

  1. Mucopolysaccharidosis type I. US National Library of Medicine website. https://ghr.nlm.nih.gov/condition/mucopolysaccharidosis-type-i. Reviewed December 2012. Published November 12, 2019. Accessed November 13, 2019.
  2. Clarke LA. Mucopolysaccharidosis type I. National Organization for Rare Disorders website. https://rarediseases.org/rare-diseases/mucopolysaccharidosis-type-i/. Published 1986. Updated 2019. Accessed October 31, 2019.
  3. Advisory Committee on Heritable Disorders in Newborns and Children. Recommended Uniform Screening Panel. Health Resources & Services Administration website. https://www.hrsa.gov/advisory-committees/heritable-disorders/rusp/index.html. Reviewed February 2019. Accessed October 31, 2019.

3 of 3

Clinical Briefings™: Diagnosing and Treating Mucopolysaccharidosis Type I: Opportunities to Improve Prognosis is a continuing medical education–certified activity in which Paul Orchard, MD, a pediatrician and expert in the field of cell therapies for inherited metabolic diseases, discusses diagnosis and treatment options when caring for patients with mucopolysaccharidosis type I (MPS I).

This online activity concludes with a discussion of current standards of treatment in MPS I and gene therapy as a promising new strategy for treating this disease. Below are highlights of the topics covered during the activity.

  • Benefits and limitations of allogeneic stem cell transplant and enzyme replacement therapy1,2
  • Emergence of gene therapy for MPS I3
  • Clinical data supporting investigational therapies that have received orphan drug product, rare pediatric disease, and fast track designations from the Food and Drug Administration for treating MPS I4,5

“There is a great deal of interest in using gene therapy as an approach for MPS I. There is an opportunity for the cells that are corrected to make potentially a 100-fold more enzyme than they would otherwise make, and that might prove more effective as a treatment.”
—Paul Orchard, MD

References

  1. Jameson E, Jones S, Remmington T. Enzyme replacement therapy with laronidase (Aldurazyme) for treating mucopolysaccharidosis type I. Cochrane Database Syst Rev. 2016;4:CD009354. doi: 10.1002/14651858.CD009354.pub4.
  2. Muenzer J, Wraith JE, Clarke LA, and the International Consensus Panel on Management and Treatment of Mucopolysaccharidosis. Mucopolysaccharidosis I: management and treatment guidelines. Pediatrics. 2009;123(1):19-29. doi: 10.1542/peds.2008-0416
  3. Fraldi A, Serafini M, Sorrentino NC, Gentner B, Aiuti A, Bernardo ME. Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches. Ital J Pediatr. 2018;44(suppl2):130. doi: 10.1186/s13052-018-0565-y.
  4. RGX-111 Gene Therapy in Patients With MPS I. https://clinicaltrials.gov/ct2/show/NCT03580083. Updated April 17, 2019. Accessed November 4, 2019.
  5. Sangamo Announces Interim Results of Phase 1/2 EMPOWERS Study Evaluating SB-318 Zinc Finger Nuclease (ZFN) In Vivo Genome Editing Demonstrating Increased Leukocyte IDUA Activity in Patients with MPS I [news release]. Brisbane, CA: Sangamo Therapeutics, Inc; February 7, 2019. https://www.prnewswire.com/news-releases/sangamo-announces-interim-results-of-phase-12-empowers-study-evaluating-sb-318-zinc-finger-nuclease-zfn-in-vivo-genome-editing-demonstrating-increased-leukocyte-idua-activity-in-patients-with-mps-i-300791647.html. Accessed November 4, 2019.

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