The Premier Family Resource for Metachromatic Leukodystrophy Information & Support since 1999!
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™…
** Member of MLD Foundation’s Medical and Scientific Advisory Board (MSAB) or the ** MLD Foundation Board (fiduciary)
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
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.