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MPS Newborn Screening Meeting


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MPS Newborn Screening Meeting Banner

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Add to Calendar MPS Newborn Screening Meeting 4/14/2025 4:30:00 PM 4/16/2025 1:30:00 PM America/Chicago For More Details: https://umncpd.cloud-cme.com/course/courseoverview?EID=6123 Description: This event is planned and hosted by the University of Minnesota's MPS Center. For more information contact Shannon Lunn at [email protected] 2025 MPS Newborn Screening (NBS) Meeting, hosted by the University of Minnesota’s MPS Center, is a unique and invaluable opportunity for professionals in the fields of genetics, pediatrics, and rare disease research. This event, scheduled from April 14-16, brin... McNamara Alumni Center and as a Live Webinar false MM/DD/YYYY


Date & Location
Monday, April 14, 2025, 4:30 PM - Wednesday, April 16, 2025, 1:30 PM CST, McNamara Alumni Center and as a Live Webinar, Minneapolis, MN

Target Audience
Professions - Laboratory Professional, Nurse, Other, Physician

Overview

This event is planned and hosted by the University of Minnesota's MPS Center. For more information contact Shannon Lunn at [email protected].

The 2025 MPS Newborn Screening (NBS) Meeting, hosted by the University of Minnesota’s MPS Center, is a unique and invaluable opportunity for professionals in the fields of genetics, pediatrics, and rare disease research. This event, scheduled from April 14-16, brings together some of the most prominent experts to explore cutting-edge advancements in mucopolysaccharidosis (MPS) and the evolving landscape of newborn screening.

Participants will gain deep insights into the critical importance of early detection in conditions like MPS I (Hurler Syndrome) and MPS II (Hunter Syndrome), which, without timely diagnosis, can rapidly progress and become life-threatening. The conference will showcase the latest innovations in gene therapies, emerging screening technologies, and clinical approaches that can significantly improve patient outcomes.

This event offers a unique opportunity to engage with the global MPS community, exchange knowledge, and help shape the future of MPS care. Whether you are a clinician, researcher, or patient advocate, the 2025 MPS NBS Meeting is a must-attend for those committed to advancing newborn screening and rare disease treatment.


Objectives
At the conclusion of this course, learners should be able to:

  1. Recognize the challenges in newborn screening for MPS disorders to ensure early and accurate diagnosis and establishment of phenotype and how this may influence interventions and improved patient outcomes.
  2. Identify the latest advancements in MPS treatments, including emerging therapies such as gene therapy, and how these may be employed in patient care strategies.
  3. Be aware of the importance of effective communication strategies and collaborative practices among multidisciplinary teams involved in the management of MPS to enhance care coordination.
  4. List key gaps in achieving optimal treatments for MPS disorders, including the role of genotype-phenotype and early detection and the risks/benefits of available treatment options.

Registration

 

Registration Policies

  • Pre-registration is requested by March 31, 2025 for in-person attendance. Late registrants cannot be guaranteed course materials, etc. On-site registration will be allowed as space permits. All on-site registrants must use their own device to submit their registration and payment by credit card via CE Portal (cash/check not accepted). Registration through CE Portal is required even if attendance is free-of-charge. 
  • Special needs, such as dietary restrictions, lactation room, etc., should be requested in advance. These requests cannot always be honored on site.
  • Cancellation policy: In the event you need to cancel your registration, the registration fee, less a $50* administrative fee, will be refunded if you notify us by 4:30 p.m. March 31, 2025, two weeks prior to the start of the conference. No refunds will be made after this date. (*the administrative fee is $15 for webinar registrations and resident/student/fellow registration categories.)
  • UMN Office of Continuing Professional Development (OCPD) and/or our partner in providing the activity reserves the right to cancel any activity due to unforeseen circumstances. In the event the activity is cancelled, registration fees will be refunded. UMN OCPD and/or our partner in providing the activity is not responsible for any travel-related costs, charges, or expenses, including but not limited to fees assessed by airline, travel, or lodging agencies.

Accreditation

In support of improving patient care, University of Minnesota, Interprofessional Continuing Education is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC) to provide continuing education for the healthcare team.

Credit Designations

American Medical Association
The University of Minnesota, Interprofessional Continuing Education designates this live activity for a maximum of 10.25 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.  

American Nurses Credentialing Center (ANCC)
This activity is awarded up to 10.25 ANCC contact hours.

American Society for Clinical Laboratory Science
P.A.C.E. ® Credit for this event will be provided by the UMN Medical Laboratory Sciences Program using the ASCLS CE Organizer. The UMN Medical Laboratory Sciences Program is approved as a provider of continuing education programs in the clinical laboratory sciences by the ASCLS P.A.C.E. ® Program. The course will offer up to 10.0 credits.

Other Healthcare Professionals
Individuals who participate in this CE activity may submit their statement of participation to their appropriate accrediting organizations or state boards for consideration of credit. The participant is responsible for determining whether this activity meets the requirements for acceptable continuing education.

 

In Person Attendance

University of Minnesota McNamara Alumni Center
200 SE Oak Street
Minneapolis, MN 55455

Directions

Nearby hotel options:

  • Days Inn Hotel by Wyndham, 2407 University Ave. SE and Washington Ave. Minneapolis, MN 55414
    612-254-6418

  • Graduate by Hilton Minneapolis, 615 Washington Ave. SE, Minneapolis, MN 55414
    612-379-8888

Live Webinar Attendance

See the "Access Course" tab above to obtain the zoom webinar link. It is only available to individuals who are registered and are signed in to their CE Portal account.



Mitigation of Relevant Financial Relationships


University of Minnesota Continuing Professional Development adheres to the ACCME’s Standards for Integrity and Independence in Accredited Continuing Education and has policies in place to ensure balance, independence, objectivity and scientific rigor in all of its educational activities. Any individuals in a position to control the content of a CE activity, including faculty, planners, reviewers or others are required to disclose all financial relationships with ineligible entities; individuals who fail to complete the disclosure in advance of the course are not eligible to be involved in this course. Specific disclosure information will be shared with learners prior to the start of the course. All relevant conflicts of interest have been mitigated prior to the commencement of the activity.

Member Information
Role in activity
Nature of Relationship(s) / Name of Ineligible Company(s)
Faculty Photos
Amy Gaviglio, MS, CGC
Connetics Consulting, LLC
Co-Director, Faculty
Nothing to disclose
Faculty Photos
Paul J Orchard, MD
Professor
University of Minnesota
Course Director, Faculty
Grant or research support-Sanofi S.A.|Independent Contractor (included contracted research)-Ionis|Other: Consultant/Clinical Research Support-Immusoft|Other: Consultant/Clinical Research Support-Bluebird Bio|Other: Consultant/Clinical Research Support-Avrobio|Other: Consultant/Clinical Research Support-Imel (Relationship has ended)|Other: Consultant/Clinical Research Support
Taralyn A Anderson
Parent of Child with MPS1
Faculty
Nothing to disclose
Natasha Bonhomme, BA
Expecting Health
Faculty
Grant or research support-Orchard Theraputics|Grant or research support-Applied Theraputics
Faculty Photos
Barbara K Burton, MD
Professor of Pediatrics Northwestern University Feinberg School of Medicine
Lurie Children's Hospital
Faculty
Consulting Fee-Takeda Pharmaceuticals (Any division)|Consulting Fee-Biomarin|Consulting Fee-Sanofi S.A.|Consulting Fee-JCR Pharma|Consulting Fee-Chiesi|Consulting Fee-Applied Therapeutics (Relationship has ended)|Consulting Fee-Alltrna|Consulting Fee-Moderna|Consulting Fee-Ultragenyx|Speakers Bureau-Biomarin|Speakers Bureau-Amgen|Grant or research support-Biomarin|Grant or research support-Takeda Pharmaceuticals (Any division)|Grant or research support-Sangamo|Grant or research support-Homology Medicines (Relationship has ended)|Grant or research support-Denali|Grant or research support-JCR Pharma|Grant or research support-Ultragenyx
Faculty Photos
Mark Dant
Executive Director
Ryan Foundation, Inc.
Faculty
Nothing to disclose
Faculty Photos
M. Christine Dorley, PhD
Division Chief
Maryland Department of Health Laboratories Administration
Faculty
Nothing to disclose
Larisa Elizondo

Faculty
Nothing to disclose
Faculty Photos
Michael Gelb, PhD
Boris and Barbara Weinstein Endowed Chair in Chemistry
Univ. of Washington
Faculty
Nothing to disclose
Faculty Photos
Samuel Goldfarb, MD
University of Minnesota
Faculty
Nothing to disclose
Faculty Photos
Jennifer Goldstein, PhD
University of North Carolina at Chapel Hill
Faculty
Nothing to disclose
Faculty Photos
Patricia Hall, PhD
Co-Director of the Biochemical Genetics Laboratory
Mayo Clinic
Faculty
Nothing to disclose
Faculty Photos
Paul Harmatz, MD
University of California, San Francisco
Faculty
Consulting Fee-Chiesi|Consulting Fee-Capsida (Relationship has ended)|Grant or research support-Sangamo|Grant or research support-Orphazyme|Grant or research support-Denali|Grant or research support-JCR|Grant or research support-RegenXbio|Grant or research support-QED|Grant or research support-Ascendis (Relationship has ended)|Grant or research support-BioMarin|Grant or research support-Takeda Pharmaceuticals (Any division)|Consulting Fee-Edigene (Relationship has ended)|Consulting Fee-Grace Science|Grant or research support-Idorsia|Consulting Fee-Novel Pharma (Relationship has ended)|Consulting Fee-Orchard Therapeutics|Grant or research support-Prevail|Consulting Fee-Renoviron (Relationship has ended)|Consulting Fee-Sialogen (Relationship has ended)|Grant or research support-Allievex (Relationship has ended)|Consulting Fee-BioMarin|Consulting Fee-Mirum Pharma (Relationship has ended)|Consulting Fee-Neurogene|Consulting Fee-Rallybio (Relationship has ended)|Consulting Fee-Sanofi|Consulting Fee-Spruce Bio|Grant or research support-Amicus (Relationship has ended)|Grant or research support-ASPA|Grant or research support-Azafaros|Grant or research support-Calcilytics|Grant or research support-Immusoft|Grant or research support-Orchard Therapeutics|Grant or research support-Sanofi
Faculty Photos
Simon A Jones, MB ChB
Consultant in Paediatric inherited metablic disease, Senior Lecturer
University of Manchester
United Kingdom
Faculty
Consulting Fee-Novartis Corporation Pharmaceuticals|Grant or research support-Sanofi S.A. (Relationship has ended)|Speakers Bureau-Chiesi (Relationship has ended)|Advisor-Lilly (Any division)
Faculty Photos
Kate S Kucera, PhD, HCLD(ABB)
Self
Faculty
Nothing to disclose
Faculty Photos
Allie Ladd
Mom of two MPS1 warriors
The Kennedy Ladd Foundation
Faculty
Nothing to disclose
Kristin McKay
Executive Director
Project Alive
Faculty
Nothing to disclose
Faculty Photos
Joseph Muenzer, MD, PhD
Distinguished Professor
University of North Carolina at Chapel Hill
Faculty
Grant or research support-Takeda Pharmaceuticals (Any division)|Consulting Fee-Takeda Pharmaceuticals (Any division)|Grant or research support-Denali|Consulting Fee-Sanofi S.A.|Consulting Fee-JCR Pharmaceuticals
Faculty Photos
Joseph Orsini, PhD
Deputy Director Newborn Screening Program
New York State Department of Health
Faculty
Nothing to disclose
Faculty Photos
Kelsey Sala-Hamrick, PhD
Assistant Professor
Eastern Michigan University
Faculty
Nothing to disclose
Faculty Photos
Dylan Simon
Director of Policy
EveryLife Foundation for Rare Diseases
Faculty
Nothing to disclose
Faculty Photos
Richard Steet, PhD
Director of Research, Head of JC Self Research Institute
Greenwood Genetic Center
Faculty
Consulting Fee-BridgeBio
Kevin Walker, MD
Gillette Children's Specialty Healthcare
Faculty
Nothing to disclose
Faculty Photos
Chester Whitley, MD, PhD
Professor
University of Minnesota
Faculty
Nothing to disclose
Zahidullah Zahid, MA
Home
Faculty
Nothing to disclose
Elizabeth Braunlin, MD, PhD
University of Minnesota
Faculty, Planning Committee Member
Nothing to disclose
Faculty Photos
Julie Beth Eisengart, PhD
Associate Professor, Pediatric Neuropsychologist
University of Minnesota
Faculty, Planning Committee Member
Speakers Bureau-Takeda Pharmaceuticals (Any division)|Consulting Fee-JCR Pharmaceuticals|Consulting Fee-Novel Therapeutics|Consulting Fee-Orchard Therapeutics (Relationship has ended)|Independent Contractor (included contracted research)-Orchard Therapeutics|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Sanofi S.A.|Grant or research support-Prevail (Relationship has ended)|Grant or research support-Lysogene (Relationship has ended)|Other: Donation of drug for research trial-Sobi (Relationship has ended)
Faculty Photos
N. Matthew Ellinwood, DVM
Chief Scientific Officer
National MPS Society
Faculty, Planning Committee Member
Honoraria-Denali Therapeutics (Relationship has ended)|Honoraria-REGENXBIO, Inc. (Relationship has ended)|Honoraria-EdiGene Biotechnology USA (Relationship has ended)|Consulting Fee-Lilly (Any division) (Relationship has ended)
Faculty Photos
Ashish Gupta, MD
MD, MPH
University of Minnesota
Faculty, Planning Committee Member
Consulting Fee-Vertex Pharmaceuticals|Grant or research support-Jazz Pharmaceuticals, Inc.|Consulting Fee-Emerging Therapies Solutions
Terri Klein, CNPM MPS
President & CEO
National MPS Society
Faculty, Planning Committee Member
Advisor-Sanofi S.A.|Advisor-Ultragenyx|Honoraria-BioMarin|Honoraria-Amicus Therapeutics (Relationship has ended)
Faculty Photos
Troy Lund, MD
University of Minnesota
Faculty, Planning Committee Member
Nothing to disclose
Faculty Photos
Nishitha R Pillai, MB BS
University of Minnesota
Faculty, Planning Committee Member
Honoraria-Sanofi S.A. (Relationship has ended)|Grant or research support-Sanofi S.A.|Consulting Fee-Denali
Jennifer A Braun (Danielson), NP
Masonic Cancer Center
Nurse Planner
Nothing to disclose
Shannon Lunn, RN
University of Minnesota
Nurse Planner
Other: Business Advisory Board Member-Parent Ready
Kate Carosella, MA
University of Minnesota
Planning Committee Member
Nothing to disclose
Kshea Hale, MPH
Senior Specialist, Newborn Screening & Genetics
Association of Public Health Laboratories
Planning Committee Member
Nothing to disclose
Patrick V Hopkins
Newborn Screening Project Specialist
Missouri State Public Health Laboratory, Newborn Screening
Planning Committee Member
Nothing to disclose
Bradley Miller, MD, PhD
Professor
University of Minnesota Medical School
Planning Committee Member
Grant or research support-Pfizer (Any division)|Advisor-Ascendis Pharma|Advisor-BioMarin|Grant or research support-Alexion Pharmaceuticals|Grant or research support-Aeterna Zentaris|Grant or research support-Lumos|Grant or research support-Sangamo|Advisor-Tolmar|Advisor-Endo Pharmaceuticals, Inc.|Advisor-Bristol-Myers Squibb Company|Grant or research support-Novo Nordisk (Any division)|Grant or research support-OPKO|Grant or research support-AbbVie (Any division)|Advisor-Eton Pharmaceuticals|Advisor-GeneScience (Relationship has ended)|Advisor-Soleno|Advisor-Amgen, Inc.|Advisor-Sanofi S.A.|Grant or research support-Foresee

Monday, April 14, 2025
 
Welcome - Moderated by Nishitha Pillai, MB, BS (This session is available to in-person attendees only.)

Social Time: Networking and informal conversations
5:00PM - 6:00PM

Plated Meal: Group dinner to kick off the event
6:00PM - 6:30PM

Access Event Recording Here - Opening Remarks and Event Overview
6:30PM - 6:35PM

Family Perspectives: The Impact of Early Diagnosis
6:35PM - 6:50PM
Larisa Elizondo

Family Perspectives: The Impact of Early Diagnosis
6:50PM - 7:05PM
Allie Ladd

Closing Remarks and Preview of Days 2 & 3
7:05PM - 7:10PM
Paul J Orchard, MD

Tuesday, April 15, 2025
 
Overview of MPS and NBS - Moderated by Paul Harmatz, MD

Breakfast
8:00AM - 8:30AM
Welcome and Opening Announcements
8:30AM - 8:35AM
Paul Harmatz, MD

Opening Address
8:35AM - 8:45AM
Mark Dant
Comprehensive overview of MPS Types, Therapies, Terminology
8:45AM - 9:15AM
Chester Whitley, MD, PhD

Testing Strategies of NY State Program and Workflow Integration for MPS I & MPS II
9:15AM - 9:30AM
Joseph Orsini, PhD

Pathways to Implementation
9:30AM - 9:45AM
Amy Gaviglio, MS, CGC

Break
9:45AM - 10:00AM

 
NBS: A Deeper Dive - Moderated by Amy Gaviglio, MS, CGC

Global importance of NBS
10:00AM - 10:20AM
Joseph Muenzer, MD, PhD
Overview of Testing Methodologies: Maryland's Experience
10:20AM - 10:40AM
M. Christine Dorley, PhD

Advancements in GAG Testing for MPS in Newborn Screening
10:40AM - 11:00AM
Michael Gelb, PhD

The ClinGen Lysosomal Diseases Variant Curation Expert Panel
11:00AM - 11:20AM
Jennifer Goldstein, PhD

MPS 2 Data Insights and Analysis
11:20AM - 11:40AM
Barbara K Burton, MD
Functional Resolution of IDUA Variants using a Cell-Based Biochemical Platform
11:40AM - 12:00PM
Richard Steet, PhD

Panel Discussion: Disease vs. No Disease – Understanding Phenotype
12:00PM - 12:30PM
Amy Gaviglio, MS, CGC (Moderator)
Michael Gelb, PhD (Panelist)
Barbara K Burton, MD (Panelist)
Richard Steet, PhD (Panelist)
Joseph Muenzer, MD, PhD
M. Christine Dorley, PhD
Jennifer Goldstein, PhD

Recognition
12:30PM - 12:35PM
Lunch
12:35PM - 1:30PM

 
NBS: Results, Notification, Policy - Moderated by Terri Klein, CNPM, MPS

Educating Providers and Effective Communication of Results
1:30PM - 1:45PM
Amy Gaviglio, MS, CGC

Family Perspectives: Receiving the News of a Positive Screen
1:45PM - 2:05PM
Taralyn A Anderson
Psychosocial Impact and Support
2:05PM - 2:20PM
Kelsey Sala-Hamrick, PhD

Newborn Screening Inequities and Policy Solutions
2:20PM - 2:35PM
Dylan Simon

The Role of Advocacy and Families in Newborn Screening
2:35PM - 2:50PM
Natasha Bonhomme, BA

Panel Discussion: Effective Strategies and Barriers in NBS for MPS
2:50PM - 3:30PM
Amy Gaviglio, MS, CGC (Panelist)
Dylan Simon (Panelist)
Natasha Bonhomme, BA (Panelist)
Terri Klein, CNPM MPS (Moderator)
Taralyn A Anderson (Panelist)
Kelsey Sala-Hamrick, PhD (Panelist)

 
Non-Credit Session

Posters and Exhibits
3:30PM - 5:00PM
Social Reception
3:30PM - 6:00PM
Wednesday, April 16, 2025
 
MPS: Phenotypes & Outcomes - Moderated by Ashish Gupta, MD

Breakfast
8:00AM - 8:30AM

Welcome and Opening Announcements
8:30AM - 8:35AM
Ashish Gupta, MD

Cardiac Considerations and Insights
8:35AM - 8:50AM
Elizabeth Braunlin, MD, PhD

Neurodevelopmental Outcomes and Implications
8:50AM - 9:05AM
Julie Beth Eisengart, PhD

Dysostosis Multiplex: Orthopedic Considerations
9:05AM - 9:20AM
Kevin Walker, MD

Airway and Pulmonary Disease Management
9:20AM - 9:35AM
Samuel Goldfarb, MD

Break
9:35AM - 9:45AM

 
Treatment Implementation - Moderated by Paul Orchard, MD

Advising Families in a Rapidly Evolving Therapeutic Landscape
9:45AM - 10:00AM
N. Matthew Ellinwood, DVM

Current Therapies: Enzyme Replacement Therapy (ERT) and Allogeneic Hematopoietic Cell Transplantation (HCT)
10:00AM - 10:15AM
Troy Lund, MD

Advances in Gene Therapy
10:15AM - 10:30AM
Simon A Jones, MB ChB

Progress in New Enzyme Development
10:30AM - 10:45AM
Paul Harmatz, MD

Panel Discussion
10:45AM - 11:05AM
Paul J Orchard, MD (Moderator)
N. Matthew Ellinwood, DVM (Panelist)
Troy Lund, MD (Panelist)
Paul Harmatz, MD (Panelist)
Simon A Jones, MB ChB (Panelist)
Break
11:05AM - 11:20AM
 
Screening for Other MPS - Moderated by Matthew Ellinwood, DVM

Family Perspectives: Advocates and Families of MPS Types II, III, IV, VI, VII
11:20AM - 11:30AM
Kristin McKay
Family Perspectives: Advocates and Families of MPS Types II, III, IV, VI, VII
11:30AM - 11:40AM
Zahidullah Zahid, MA
Multiplexing MPS Disorders
11:40AM - 11:50AM
Joseph Orsini, PhD

GAG NRE NBS Data
11:50AM - 12:05PM
Patricia Hall, PhD
Whole Genome Sequencing in NBS
12:05PM - 12:20PM
Kate S Kucera, PhD, HCLD(ABB)

Panel Discussion: Expanding Screening to Other MPS Types
12:20PM - 12:50PM
Joseph Orsini, PhD (Panelist)
N. Matthew Ellinwood, DVM (Moderator)
Zahidullah Zahid, MA (Panelist)
Kristin McKay (Panelist)
Kate S Kucera, PhD, HCLD(ABB) (Panelist)
Patricia Hall, PhD (Panelist)

Boxed Lunch
12:50PM - 1:30PM

If you are a confirmed exhibitor with questions about the meeting, contact Shannon Lunn.


Webinar Access for MPS Newborn Screening Meeting

Individuals who have registered for this course can access the zoom webinar link here. If you are seeing the "Register" button it is because you either have not registered or you are not signed into your CE Portal account; please sign in to continue. 

Note the webinar link is for Tuesday, April 15 and Wednesday, April 16. A webinar link is not available for Monday, April 14 due to the social nature of the content. 

Register

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Minneapolis, MN 55455

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