AGENDA - 2023 MLD Family Conference®

Tentative Schedule – Subject to change – All times EDT (New York)

Friday, July 14th

9:00 AM

Registration Opens

Children’s Program/Respite Room opens

Light Continental breakfast

9:30 

Welcome & Introductions

Dean Suhr, President, MLD Foundation *

Teryn Suhr, Executive Director, MLD Foundation *

10:00

The Winding Road From Bench to Trials to Regulatory Approval

  • MLD and its Relationship to the Lysosome
  • Natural History & Data Collection
  • Genotype/Phenotype
  • Clinical Trials
  • Regulatory Engagement, Review & Approvals

facilitated by
Cara M. Weismann PhD, U Penn Orphan Disease Center

Laura Adang MD PhD, Children’s Hospital of Philadelphia
Michael Gelb PhD, Univ WA
Paul Orchard MD, U MN
Marc Patterson  MD, Mayo Clinic **

10:45

Break

11:00

BioPharma Updates on Research/Therapies

  • Orchard Therapeutics
  • Homology Medicines
  • Passage Bio
  • Affinia Therapeutics
  • Takeda Pharmaceuticals
  • Sanofi 

11:45

Where Do We Go From Here? … The DTAR 3-Legged Stool

Laura Adang MD PhD, Children’s Hospital of Philadelphia **
Michael Gelb PhD, Univ WA
Paul Orchard MD, U MN
Marc Patterson  MD, Mayo Clinic **

12:30

Lunch
(Friday Industry and academic experts photo)

2:00

MLD Newborn Screening Academy … Identifying Individuals When Therapies are Effective

Tara Mathiesen RN, Newborn Screening & Diagnostics Expert
Laura Adang MD  PhD, Children’s Hospital of Philadelphia **
Michael Gelb PhD, Univ WA
Marc Patterson  MD, Mayo Clinic **
Public Heath Official (invited)

3:00

Therapies Are Of No Value Without Access & Reimbursement

Jay Greissing, Washington DC Policy Team, Biomarin
Legislative Policy Expert (invited)

3:30

MLD Advocacy Academy – What’s Your story?

  • My Story
  • Your Story

Krystal Meyers, Patient Advocate & MLD Mom

BioPharma Policy Expert (invited)
Legislative Policy Expert (invited)

3:30

Exploring Communication /ACC  & AT Devices

Sabine Leclaire, LSP, LTVEC

4:15

Break

4:30

Ladies Breakout & Men’s Breakout – A time for those new and for those more experienced on the MLD journey to come together in a small, caring, confidential environment

Academia & Industry Breakout – brainstorm of priorities for future MLD research, clinical, and advocacy efforts

5:30

Family Time

6:30

Dinner

9pm – 12

After Hours with the MLD Family™… 

Saturday, July 15th 

8:30

 

Buffet Breakfast

9:00

Registration opens

Children’s Program/Respite Room opens

9:30

MLD Advocacy Academy – How We Can & Are Making  Difference

  • My Story
  • Your Story

Krystal & Kaden Meyers, Patient Advocate & MLD Mom

Jay Greissing, JD BioPharma Policy Expert

10:15

Meeting the Needs of Siblings

Jenni Rogers MS, CCLS, Child Life Specialist

11:00

Break

11:15

Group Photo

11:45

Lunch

1:15

Remembering Our MLD Angels – Butterfly Ceremony – A special remembrance of those who have earned their Butterfly Wings since our last time together (2019)… and honoring all of our MLD Angels

All attendees … we will live stream the ceremony

2:15

Break

2:30

Understanding Grief

Kendra Bjoraker, PhD, Pediatric Neuropsychologist

3:15

Making Memories, Creating  A Legacy

Jenni Rogers MS, CCLS, Child Life Specialist

4:15

The Gift of Inclusion

Pat Lang, mother to angel Madigan

5:15

Wrap Up/Group Hug

Everyone

5:30

Family time

6:45

Celebration Parade – call time

Everyone

7:00

reaching for the Stars Celebration Parade, Closing Dinner Party  

Everyone

10-12

After Hours with the MLD Family™… 

Thank you to our SPONSORS, DONORS, & GRANT PROVIDERS

Thank you to our generous sponsors and grant providers who through their generous support make this conference much more affordable for the attending families. We anticipate and want to thank in advance the families who individually donated to support of our conference scholarship fund.

Thank you to our SPONSORS, DONORS, & GRANT PROVIDERS

Takeda Pharmaceuticals logo - 2023
Rock On Flinchum logo

Thank you to our generous sponsors and grant providers who through their generous support make this conference much more affordable for the attending families. We anticipate and want to thank in advance the families who individually donated to support of our conference scholarship fund.