Hunter’s Hope Foundation

2022 Global Virtual Leukodystrophy Symposium Program

Please note, this program is subject to change and will be finalized a week prior to the meeting.

*Families are welcome to attend all sessions.

Medical Symposium: Day One

Zoom Webinar Link:

Leukodystrophy Families Welcome 

Tuesday, July 19, 2022 – 1:00pm – 5:00pm ET

Newborn Screening (NBS) for Leukodystrophies

1:00pm ET — Welcome and Overview, Presented by: Anna Grantham

Leukodystrophy NBS State Updates, Moderated by: Joseph Orsini, PhD

1:10 – 1:15pm ET — New York, Presented by: Joseph Orsini, PhD

1:15 – 1:20pm ET — Missouri, Presented by: Tracy Klug

1:20 – 1:25pm ET — Kentucky, Presented by: Darrin Sevier

1:25 – 1:30pm ET — Ohio, Presented by: Margie Ream, MD, PhD

1:30 – 1:35pm ET — Tennessee, Presented by: M. Christine Dorley, PhD

1:35 – 1:40pm ET — Illinois, Presented by Joseph Orsini, PhD

1:40 – 1:45pm ET — New Jersey, Presented by Miriam Schachter, PhD and Caitlin Vail

1:45 – 1:50pm ET — Indiana, Presented by Barb Lesko, MT (ASCP)

1:50 – 1:55pm ET — Pennsylvania, Presented by: Patricia Alexander, RN 

1:55 – 2:10pm ET — Discussion


NBS for Leukodystrophies, Moderated by: Margie Ream, MD, PhD

2:10 – 2:30pm ET — NBS for Krabbe Disease (KD) – Updates and Improvements Presented by: Dietrich Matern, MD, PhD

2:30 – 2:45pm ET — KD NBS Advisory Council, Presented by: Joanne Kurtzberg, MD

2:45 – 3:00pm ET — KD NBS Family Guide, Presented by: Robert Thompson Stone, MD

3:00 – 3:15pm ET — Patient Experiences, KD NBS

3:15 – 3:30pm ET — Improving KD NBS Patient Experiences and Outcomes,
Presented by: Joanne Kurtzberg, MD

3:30 – 3:45pm ET — KD NBS Outcomes – Standardizing Nerve Conduction, Presented by Margie Ream, MD, PhD and Joanne Kurtzberg, MD

3:45 – 4:00pm ET — Biochemical NBS meets DNA Sequencing NBS: It is not one or the other, Presented by: Michael Gelb, PhD

4:00 – 4:15pm ET — ALD NBS Overview/Update, Presented by: Ali Fatemi, MD and Eric Mallack, MD

4:15 – 4:30pm ET — MLD NBS Overview/Update, Presented by: Michael Gelb, PhD

4:30 – 4:45pm ET — CTX NBS Overview/Update, Andrea DeBarber, PhD

4:45 – 5:00pm ET — Discussion

Medical Symposium: Day Two

Zoom Webinar Link:

Leukodystrophy Families Welcome

Wednesday, July 20, 2022 – 1:00pm – 5:00pm ET

Leukodystrophy Clinical Care and Treatments, moderated by Robert Thompson Stone, MD

1:00pm ET — Welcome and Overview, Presented by: Anna Grantham

1:05 – 1:20pm ET — Leukodystrophy Care Network Overview

Industry Updates: Leukodystrophy Clinical Trials and Research

1:20 – 1:40pm ET — Passage Bio, Presented by: Samiah Al-Zaidy, MD

1:40 – 2:00pm ET — Forge Biologics, Presented by: Maria Escolar, MD

2:00 – 2:20pm ET — Travere Therapeutics, Presented by: Amy Ponte, PhD

2:20 – 2:40pm ET — Orchard Therapeutics, Presented by: Angela Tom, MS, FNP-C

2:40 – 3:00pm ET — Vigil Neuroscience, Inc, Presented by: Andreas Meier, MD

3:00 – 3:15pm ET — Discussion

3:15 – 3:35pm ET — DUOC: A Cord Blood Therapy to Myelinate the Brain,
Presented by: Joanne Kurtzberg, MD

3:35 – 3:50pm ET —  Early Signs and Symptoms of Leukodystrophies: A Case Based Guide, Presented by: Maura Ruzhnikov, MD, FACMG

3:50 – 4:05pm ET — A Systematic Evidence Review for Cerebrotendinous Xanthomatosis: Phenotypic Spectrum of Disease and Response to Therapy, Presented by: Austin Larson, MD

4:05 – 4:20pm ET — Update on Rehabilitation for Children with Leukodystrophies,
Presented by: Sally Evans, MD

4:20 – 4:35pm ET — Respiratory Clinical Practice Guidelines for Leukodystrophies, Presented by: Geovanny Perez, MD

4:35 – 4:50pm ET — Clinical Care for Patients with Cerebrotendinous Xanthomatosis, Presented by: Brian Wishart, MD

4:50 – 5:00pm ET — Discussion

To learn more about our individual LCN Centers, we invite you to watch their presentations located on each center’s page –
click here.

Family Symposium: Day One

Wednesday, July 20, 2022 – 7:00pm ET

Family Introductions

Zoom Meeting Link:

Each registered family in attendance will have a chance to introduce themselves and share their story.
*All are welcome to attend.

Family Symposium: Day Two

Thursday, July 21, 2022 – 6:00pm – 7:30pm ET

LCN/NBS Advocacy & Family Programs

Zoom Meeting Link:

6:00 – 6:30pm ET — LCN & NBS Updates/Advocacy, Presented by: Anna Grantham

6:30 – 7:00pm ET — Family Programs/Resources, Presented by: Melissa Jablonski

7:00 – 7:30pm ET — Discussion

Family Symposium: Day Three

Zoom Webinar Link:

Friday, July 22, 2022 – 9:00am – 12:30pm ET

A Day of Hope & Inspiration for Families

9:00 – 10:00am ET — The Heart of the Caregiver, From Overwhelmed to Overjoyed, Presented by: Mary Tutterow

10:00 – 11:00am ET — Imagine Heaven, Presented by: John Burke

11:00 – 11:20am ET — RightNow Media, Presented by: Conzie Wells

11:20 – 11:40am ET — Pure Flix, Presented by: Krista Scott

11:40 – 12:00pm ET — Light your Heart with Hope Podcast, Presented by: Jacque Waggoner & Erin Kelly Bean

12:00pm – 12:30pm ET — Story Time with Jill, Presented by: Jill Kelly


Men & Women Sessions

Zoom Meeting Link:

* These sessions are reserved for parents of an affected child.

Men’s Session — Moderated by: Mel Sereno

Women’s Session — Moderated by: Terry Hammonds

2:00 – 4:00pm ET