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Mucopolysaccharidosis Newborn Screening Meeting


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Mucopolysaccharidosis Newborn Screening Meeting Banner

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Add to Calendar Mucopolysaccharidosis Newborn Screening Meeting 4/10/2023 4:00:00 PM 4/12/2023 1:30:00 PM America/Chicago For More Details: https://umncpd.cloud-cme.com/course/courseoverview?EID=2012 Description: Format:Hybrid (In-Person Attendance or Live Webinar)This course is planned and hosted by University of Minnesota Pediatric BMT & CT in collaboration with the National MPS Society and APHL.The MPS Center at the University of Minnesota will be hosting the 2023 MPS NBS Meeting to share the latest information on mucopolysaccharidosis and the importance of early detection through newborn screening. Earl... In Person at McNamara Alumni and as a Live Webinar false MM/DD/YYYY


Date & Location
Monday, April 10, 2023, 4:00 PM - Wednesday, April 12, 2023, 1:30 PM CST, In Person at McNamara Alumni and as a Live Webinar, Minneapolis, MN

Overview

Format: Hybrid (In-Person Attendance or Live Webinar)

This course is planned and hosted by University of Minnesota Pediatric BMT & CT in collaboration with the National MPS Society and APHL.

The MPS Center at the University of Minnesota will be hosting the 2023 MPS NBS Meeting to share the latest information on mucopolysaccharidosis and the importance of early detection through newborn screening. Early detection is critical to saving lives from the rapidly progressing rare diseases known as MPS I (Hurler Syndrome) and MPS II (Hunter Syndrome), and other subtypes.

View the Agenda/Schedule tab for the agenda. Times listed reflect CDT. 

The poster session is available to in-person attendees only.


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

  1. Discuss methods to identify pseudodeficiencies and other inconclusive test results.
  2. Identify the optimal timing, benefits, and limitations of therapies for MPS (i.e. enzyme therapy, allogeneic transplant, and gene therapy).
  3. Describe methods used to correlate test results with potential phenotypes.
  4. Identify modalities used to assess MPS (i.e. neuropsychology, cardiology, orthopedics, imaging, labs, etc.).
  5. Describe potential findings and surveillance recommendations.
  6. Identify areas of interest for future research.

Registration

In-Person Registration is now closed. There is NOT a walk-in option.

Webinar Registration Rates:

  • Physician $50
  • Other Healthcare Professional  $50
  • Resident/Fellow/Student $5
  • Medical Industry Professional $50
  • State/Territory Lab Rep $50
  • Families $0

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.

American Medical Association (AMA)
The University of Minnesota, Interprofessional Continuing Education designates this live activity for a maximum of 10.0 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 10.0 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 credits.

Other Healthcare Professionals
Other healthcare professionals who participate in this CE activity may submit their certificate 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.


Additional Information

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, 2023. 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.)

Note: This event is supported by the Health Resources and Services Administration (HRSA) of the US Department of Health and Human Services (HHS) as part of an award totaling $1,500,000 with 0% financed with non-governmental sources. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by HRSA, HHS or the US Government. For more information, please visit HRSA.gov.

All activities will take place at:
University of Minnesota McNamara Alumni Center
200 SE Oak Street 
Minneapolis, MN 55455

Map

You can also opt to register to participate via live webinar. The webinar connection information will be shared with those registered to attend approximately one week prior to the start of the course.

Hotel Information:

A block of rooms is being held at The Graduate Hotel.  The deadline to reserve a room at the reduced rate is Monday, March 13.  Use this link to reserve a room at a reduced rate.

To use the booking link:

  • Select the exact dates of your stay (top of page)
  • Click on check availability
  • Select room type - "Book now"
  • Confirm your stay, enter your name and credit card

If you prefer, you can call (612) 379-8888 and ask for a room with the group code 041023PEDI. 

Rooms are available for Monday, April 10 and Tuesday, April 11 (departure date April 12).  The Graduate is less than two blocks to the conference venue.

Any room changes or cancellations must be made 24 hours before check-in: you are responsible for the cancellation process and any fees as outlined in your confirmation.



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)
Sarah Jutila Peterson, CPCO, CPMA, CHFP
University of Minnesota
Activity Administrator
Nothing to disclose
Faculty Photos
Paul J Orchard, MD
Professor
University of Minnesota
Course Director, Faculty
Grant or research support-Sanofi S.A.|Other: Consultant/Clinical Research Support-Orchard Therapeutics|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|Other: Consultant/Clinical Research Support
Faculty Photos
Khaja Basheeruddin, PhD
Public Service Administrator
Illinois Department of Public Health
Faculty
Nothing to disclose
Susan A Berry, MD
University of Minnesota
Faculty
Nothing to disclose
Natasha Bonhomme, BA
Expecting Health
Faculty
Nothing to disclose
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. (Relationship has ended)|Consulting Fee-JCR Pharma|Consulting Fee-Moderna|Consulting Fee-Ultragenyx|Speakers Bureau-Biomarin|Speakers Bureau-Horizon|Grant or research support-Biomarin|Grant or research support-Takeda Pharmaceuticals (Any division)|Grant or research support-Sangamo|Grant or research support-Homology Medicines|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
Patricia Dickson, MD
Washington University School of Medicine
Faculty
Grant or research support-Sanofi S.A.|Grant or research support-M6P Therapeutics|Grant or research support-Alnylam|Advisor-Mandos Health
Faculty Photos
Michael Gelb, PhD
Boris and Barbara Weinstein Endowed Chair in Chemistry
Univ. of Washington
Faculty
Nothing to disclose
Scott D Grosse, PhD
Adjunct Asst Professor
University of Minnesota
Faculty
Nothing to disclose
Faculty Photos
Paul Harmatz, MD
University of California, San Francisco
Faculty
Consulting Fee-Chiesi|Consulting Fee-Inventiva|Consulting Fee-Aeglea (Relationship has ended)|Consulting Fee-Homology|Consulting Fee-Audentes (Relationship has ended)|Consulting Fee-Paradigm|Consulting Fee-JCR (Relationship has ended)|Consulting Fee-Capsida|Grant or research support-Genzyme (Any division) (Relationship has ended)|Grant or research support-Sangamo|Grant or research support-Orphazyme|Grant or research support-Inventiva (Relationship has ended)|Grant or research support-Enzyvant (Relationship has ended)|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|Grant or research support-BioMarin|Grant or research support-Takeda Pharmaceuticals (Any division)|Consulting Fee-Avrobio (Relationship has ended)|Consulting Fee-Edigene (Relationship has ended)|Consulting Fee-Grace Science|Grant or research support-Idorsia|Consulting Fee-Novel Pharma|Consulting Fee-Orchard Therapeutics|Grant or research support-Prevail|Consulting Fee-Renoviron|Consulting Fee-Sialogen|Grant or research support-SOBI (Relationship has ended)|Grant or research support-Allievex
Faculty Photos
Nicole Kelly, MPH
Research Program Manager
Children's Hospital at Montefiore/Albert Einstein College of Medicine
Faculty
Nothing to disclose
Faculty Photos
Tracy Klug
Newborn Screening Chief
Missouri Department of Health and Senior Services
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
Nishitha R Pillai, MB BS
University of Minnesota
Faculty
Honoraria-Sanofi S.A. (Relationship has ended)
Faculty Photos
Laura Pollard, PhD
Director, Biochemical Genetics Laboratory
Greenwood Genetic Center
Faculty
Nothing to disclose
Andreas Rohrwasser, Phd, MBA
Director
Utah Public Health Laboratory; University of Utah
Faculty
Nothing to disclose
Faculty Photos
Dylan Simon
Director of Policy
EveryLife Foundation for Rare Diseases
Faculty
Nothing to disclose
Faculty Photos
Kim Stephens, PhD
Executive Director
University of North Carolina
Faculty
Grant or research support-Takeda Pharmaceuticals (Any division)|Consulting Fee-Denali|Speakers Bureau-Denali|Consulting Fee-RegenXBio|Speakers Bureau-RegenXBio|Royalties or Patent Beneficiary-JCR (Relationship has ended)
Faculty Photos
Nicolas L Szabo-Fresnais, PhD
Utah DHHS
Faculty
Nothing to disclose
Faculty Photos
Silvia Tortorelli, MD, PhD
Mayo Clinic
Faculty
Nothing to disclose
Robert Wynn, MD, Director Blood and Marrow Transplant Programme, Royal Manchester Children's Hospital
Director, Pediatric Bone Marrow Transplant Program
Royal Manchester Children's Hospital
United Kingdom
Faculty
Nothing to disclose
Elizabeth Braunlin, MD, PhD
University of Minnesota
Faculty, Planning Committee Member
Grant or research support-ReGenyX Bio (Relationship has ended)
Faculty Photos
Julie Beth Eisengart, PhD
Associate Professor, Pediatric Neuropsychologist
University of Minnesota
Faculty, Planning Committee Member
Consulting Fee-Takeda Pharmaceuticals (Any division)|Consulting Fee-JCR Pharmaceuticals|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Novel Therapeutics|Consulting Fee-Orchard Therapeutics|Independent Contractor (included contracted research)-Orchard Therapeutics (Relationship has ended)|Consulting Fee-RegenxBio|Advisor-Denali Therapeutics|Membership on Advisory Committees or Review Panels, Board Membership, etc.-Sanofi S.A.
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)
Faculty Photos
Amy Gaviglio, MS, CGC
Connetics Consulting, LLC
Faculty, Planning Committee Member
Consulting Fee-Orchard Therapeutics|Honoraria-Worldwide Clinical Trials (Relationship has ended)
Kshea Hale, MPH
Senior Specialist, Newborn Screening & Genetics
Association of Public Health Laboratories
Faculty, Planning Committee Member
Nothing to disclose
Terri Klein, CNPM MPS
President & CEO
National MPS Society
Faculty, Planning Committee Member
Advisor-Sanofi S.A.|Advisor-Ultragenyx|Honoraria-BioMarin
Faculty Photos
Troy Lund, MD
University of Minnesota
Faculty, Planning Committee Member
Advisor-Sanofi S.A.
Bradley Miller, MD, PhD
Professor
University of Minnesota Medical School
Faculty, Planning Committee Member
Grant or research support-Pfizer (Any division)|Grant or research support-Orchard Therapeutics (Relationship has ended)|Advisor-Ascendis Pharma|Advisor-BioMarin|Advisor-EMD Serono|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-Vertice (Relationship has ended)
Jennifer A Braun (Danielson), NP
Masonic Cancer Center
Nurse Planner
Nothing to disclose
Faculty Photos
George Dizikes, PhD, HCLD/CC(ABB)
Knoxville Regional Laboratory Director
Tennessee Department of Health
Planning Committee Member
Nothing to disclose
Faculty Photos
Ashish Gupta, MD
MD, MPH
University of Minnesota
Planning Committee Member
Membership on Advisory Committees or Review Panels, Board Membership, etc.-Bluerock Therapeutics|Consulting Fee-Vertex Pharmaceuticals
Jasmine Hammer, PhD
UMN
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
Faculty Photos
Sikha Singh, MHS
APHL
Planning Committee Member
Nothing to disclose
Lisa Spofford, MA
University of Minnesota
Planning Committee Member
Nothing to disclose

Monday, April 10, 2023
Welcome - social hour (Non-CE Credit Event)
5:30PM - 6:30PM
Buffet and Seating (Non-CE Credit Event)
6:30PM - 7:15PM
Welcome
7:15PM - 7:20PM
Julie Beth Eisengart, PhD
Terri Klein, CNPM MPS
Program: Family Experiences, Effect of Early Diagnosis (Non-CE Credit Event) - Family Speakers
7:20PM - 7:40PM
Wrap up, what to expect on days 2 and 3 (Non-CE Credit Event)
7:40PM - 7:50PM
Julie Beth Eisengart, PhD
Tuesday, April 11, 2023
Breakfast
8:00AM - 8:30AM
Welcoming Remarks
8:30AM - 8:35AM
Paul J Orchard, MD
Session 1 - Overview of MPS and NBS: Opening Address
8:35AM - 8:45AM
Mark Dant
N. Matthew Ellinwood, DVM (Moderator)
Session 1 - Overview of MPS and NBS: Comprehensive Overview of MPS Types, Therapies & Terminology
8:45AM - 9:15AM
Joseph Muenzer, MD, PhD
N. Matthew Ellinwood, DVM (Moderator)
Session 1 - Overview of MPS and NBS: State of the states - who's using what; MPS I, MPS II - integrated
9:15AM - 9:30AM
Kshea Hale, MPH
N. Matthew Ellinwood, DVM (Moderator)
Session 1 - Overview of MPS and NBS: What can be accomplished by NBS; work to be done
9:30AM - 9:45AM
Barbara K Burton, MD
N. Matthew Ellinwood, DVM (Moderator)
Break
9:45AM - 10:00AM
Session 2 - NBS: A deeper dive: State experiences, MO, IL & NY
10:00AM - 10:30AM
Tracy Klug
Amy Gaviglio, MS, CGC (Moderator)
Joseph Orsini, PhD
Khaja Basheeruddin, PhD
Session 2 - NBS: A deeper dive: Overview of testing methodology
10:30AM - 10:45AM
Michael Gelb, PhD
Amy Gaviglio, MS, CGC (Moderator)
Session 2 - NBS: A deeper dive: GAG testing as a 2nd tier test
10:45AM - 11:00AM
Silvia Tortorelli, MD, PhD
Amy Gaviglio, MS, CGC (Moderator)
Session 2 - NBS: A deeper dive: Potential for multiplexing in MPS
11:00AM - 11:15AM
Andreas Rohrwasser, Phd, MBA
Nicolas L Szabo-Fresnais, PhD
Session 2 - NBS: A deeper dive: Molecular testing; pseudodeficiencies and other challenges
11:15AM - 11:30AM
Amy Gaviglio, MS, CGC (Moderator)
Laura Pollard, PhD
Session 2 - NBS: A deeper dive: Clinical Diagnostic Dilemmas: Case Presentation
11:30AM - 11:45AM
Troy Lund, MD
Amy Gaviglio, MS, CGC (Moderator)
Session 2 - NBS: A deeper dive: Panel Discussion: Disease or no disease? Phenotype?
11:45AM - 12:15PM
Michael Gelb, PhD (Panelist)
Silvia Tortorelli, MD, PhD (Panelist)
Andreas Rohrwasser, Phd, MBA (Panelist)
Nicolas L Szabo-Fresnais, PhD (Panelist)
Troy Lund, MD (Panelist)
Amy Gaviglio, MS, CGC (Moderator)
Laura Pollard, PhD (Panelist)
Lunch
12:20PM - 1:30PM
Session 3: NBS: Results, Notification, Policy: First visit: when, how and what should happen?
1:30PM - 1:45PM
Terri Klein, CNPM MPS (Moderator)
Susan A Berry, MD
Session 3: NBS: Results, Notification, Policy: The family experience: Case presentations - Family Speakers
1:45PM - 2:05PM
Terri Klein, CNPM MPS (Moderator)
Session 3: NBS: Results, Notification, Policy: Educating providers: what do they need to know in calling families?
2:05PM - 2:20PM
Terri Klein, CNPM MPS (Moderator)
Amy Gaviglio, MS, CGC
Session 3: NBS: Results, Notification, Policy: NBS Inequity, Policy Initiatives
2:20PM - 2:35PM
Terri Klein, CNPM MPS (Moderator)
Dylan Simon
Session 3: NBS: Results, Notification, Policy: The role of advocacy and families in NBS
2:35PM - 2:50PM
Natasha Bonhomme, BA
Session 3: NBS: Results, Notification, Policy: Panel Discussion: What works and doesn't in NBS for MPS - Family Speakers (Panelists)
2:50PM - 3:30PM
Terri Klein, CNPM MPS (Moderator)
Natasha Bonhomme, BA (Panelist)
Amy Gaviglio, MS, CGC (Panelist)
Dylan Simon (Panelist)
Poster Session (In-person only)
3:30PM - 5:00PM
Cocktail Hour
4:00PM - 6:00PM
Wednesday, April 12, 2023
Breakfast
8:00AM - 8:30AM
Welcoming Remarks
8:30AM - 8:35AM
Paul J Orchard, MD
Session 4: MPS; Phenotypes & Outcomes: Advising families in a rapidly evolving therapeutic landscape
8:35AM - 8:50AM
N. Matthew Ellinwood, DVM
Patricia Dickson, MD (Moderator)
Session 4: MPS; Phenotypes & Outcomes: Neurodevelopmental outcomes
8:50AM - 9:05AM
Julie Beth Eisengart, PhD
Patricia Dickson, MD (Moderator)
Session 4: MPS; Phenotypes & Outcomes: Dysostosis multiplex
9:05AM - 9:20AM
Bradley Miller, MD, PhD
Patricia Dickson, MD (Moderator)
Session 4: MPS; Phenotypes & Outcomes: Cardiac disease
9:20AM - 9:35AM
Elizabeth Braunlin, MD, PhD
Patricia Dickson, MD (Moderator)
Break
9:35AM - 9:45AM
Session 5: Treatment Implementation: Existing therapy: ERT
9:45AM - 10:05AM
Paul J Orchard, MD (Moderator)
Nishitha R Pillai, MB BS
Session 5: Treatment Implementation: Existing therapy: allogeneic HCT
10:05AM - 10:25AM
Paul J Orchard, MD (Moderator)
Troy Lund, MD
Session 5: Treatment Implementation: Gene therapy
10:25AM - 10:45AM
Paul J Orchard, MD (Moderator)
Robert Wynn, MD, Director Blood and Marrow Transplant Programme, Royal Manchester Children's Hospital
Session 5: Treatment Implementation: New enzyme development
10:45AM - 11:05AM
Paul J Orchard, MD (Moderator)
Paul Harmatz, MD
Break
11:05AM - 11:15AM
Session 6: Screening for other MPS: Opportunities in NBS for other MPS disorders
11:15AM - 11:25AM
N. Matthew Ellinwood, DVM (Moderator)
Kim Stephens, PhD
Session 6: Screening for other MPS: Family Perspective: II, III, IV, VI, VII advocates/families - Family Speakers
11:25AM - 11:50AM
N. Matthew Ellinwood, DVM (Moderator)
Session 6: Screening for other MPS: Feasibility of Testing
11:50AM - 12:05PM
N. Matthew Ellinwood, DVM (Moderator)
Nicole Kelly, MPH
Session 6: Screening for other MPS: Assessing the Potential Population Health Impact of Newborn Screening for MPS Disorders
12:05PM - 12:20PM
N. Matthew Ellinwood, DVM (Moderator)
Scott D Grosse, PhD
Session 6: Screening for other MPS: Panel Discussion - Family Speakers (Panelists)
12:20PM - 12:50PM
N. Matthew Ellinwood, DVM (Moderator)
Nicole Kelly, MPH (Panelist)
Scott D Grosse, PhD (Panelist)
Kim Stephens, PhD (Panelist)
Farewell Lunch/Adjourn
12:50PM - 1:30PM

We would like to thank our exhibitors:

Gold Level
Association of Public Health Laboratories

Silver Level
Novel Pharma
Waters Corporation

Bronze Level
Denali Therapeutics
Fairview Pharmacy - Home Infusion and Specialty Pharmacy
Homology Medicines
Immusoft Corporation
JCR Pharmaceuticals Co., Ltd.
Orchard Therapeutics
Paradigm Biopharma
PerkinElmer
REGENXBIO


Webinar Access for Mucopolysaccharidosis Newborn Screening Meeting
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