2022 GLIA Scientific Meeting Schedule

2022 GLIA SCIENTIFIC MEETING – DAY 1 Monday, October 10 — 8:00 a.m. to 5:00 p.m.

8:00 - 8:30 a.m.

Breakfast

8:30 - 9:00 a.m.

Welcome and Introductions Speakers: Adeline Vanderver, MD, Brenda Banwell, MD, Susan Furth, MD, PhD

Session I 9:00 - 9:45 a.m.

Aicardi-Goutières Syndrome Workgroup Leader: Laura Adang, MD, PhD (Children’s Hospital of Philadelphia) Speaker No. 1: Francesco Gavazzi, MD, PhD (Children’s Hospital of Philadelphia) Speaker No. 2: Markus Hofer, MD (University of Sydney)

9:45 - 10:30 a.m.

Canavan Disease Workgroup Leader: Florian Eichler, MD (Massachusetts General Hospital) Speaker No. 1: Annette Bley, MD (University Medical Center Hamburg) Speaker No. 2: Dominic Gessler, MD, PhD (University of Minnesota)

10:30 - 10:45 a.m.

Coffee Break

Session II 10:45 - 11:30 a.m.

Krabbe Disease Workgroup Leader: Gustavo Maegawa, MD, PhD (Columbia University) Speaker No. 1: Allison Bradbury, MS, PhD (Nationwide Children’s Hospital) Speaker No. 2: Daesung Shin, PhD (University at Buffalo)

11:30 a.m. - 12:15 p.m.

Multiple Sulfatase Deficiency (MSD) Workgroup Leader: Rebecca Ahrens-Nicklas, MD, PhD (Children’s Hospital of Philadelphia) Speaker No. 1: Lars Schlotawa, MD (University Medical Center Goettingen) Speaker No. 2: Laura Adang, MD, PhD (Children’s Hospital of Philadelphia)

12:15 - 1:00 p.m.

Lunch

Session III 1:00 - 1:45p.m.

Adrenoleukodystrophy (ALD) Workgroup Leader: Ali Fatemi, MD, MBA (Kennedy Krieger Institute) Speaker No. 1: Christine Duncan, MD (Boston Children’s Hospital) Speaker No. 2: Marc Engelen, MD, PhD (Amsterdam University Medical Centers)

1:45 - 2:30 p.m.

Alexander Disease Workgroup Leader: Amy Waldman, MD, MSCE (Children’s Hospital of Philadelphia) Speaker No. 1: Davide Tonduti, MD, PhD (Ospedale dei Bambini V. Buzzi) Speaker No. 2: Tracy Hagemann, PhD (University of Wisconsin - Madison)

2:30 - 2:45 p.m.

Coffee Break

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Session IV 2:45 - 3:30 p.m.

Hypomyelination with Brainstem and Spinal Cord Involvement and Leg Spasticity (HBSL) and Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation (LBSL) Workgroup Leader: Amena Smith-Fine, MD, PhD (Kennedy Krieger Institute) Speaker No. 1: Beth Frigola McGinn, BA (Cure LBSL) Speaker No. 2: Christina Mertz, PhD (Kennedy Krieger Institute)

3:30 - 4:15 p.m.

Metachromatic Leukodystrophy (MLD) Workgroup Leader: Samuel Gröschel, MD, PhD (University of Tübingen) Speaker No. 1: Caroline Sevin, MD, PhD (INSERM) Speaker No. 2: Nicole Wolf, MD, PhD (Amsterdam University Medical Centers)

4:15 - 5:00 p.m.

Vanishing White Matter Disease (VWM) Workgroup Leader: Marjo van der Knaap, MD, PhD (Amsterdam University Medical Centers) Speaker No. 1: Marianna Bugiani, MD, PhD (Amsterdam University Medical Centers) Speaker No. 2: Paul Tesar, PhD (Case Western Reserve University School of Medicine)

5:00 p.m.

Announcements and Wrap-Up (Day 1)

2022 GLIA SCIENTIFIC MEETING NETWORKING EVENT Monday, October 10 — 6:00 p.m. to 9:00 p.m.

Please join us after the meeting for food and drinks at the Pennsylvania Horticultural Society (PHS) Pop Up Garden on South Street! It is a short bus, carshare, or taxi ride from the Children’s Hospital of Philadelphia campus, and a perfect opportunity to catch up with old colleagues and new acquaintances after a long day of presentations. No reservations are necessary.

Time: Location: Address:

6:00 - 9:00 p.m.

PHS Pop Up Garden 1438 South Street, Philadelphia, PA 19146

NOTE: In the event of inclement weather, the networking event will be moved to an indoor location at Royal Boucherie, a lively French-inspired brasserie located in Philadelphia’s Old City neighborhood at 52 South 2nd Street, Philadelphia, PA 19106.

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2022 GLIA SCIENTIFIC MEETING – DAY 2 Tuesday, October 11 — 8:00 a.m. to 4:30 p.m.

8:00 - 8:30 a.m.

Breakfast

Session V 8:30 - 9:15 a.m.

Pelizaeus Merzbacher Disease (PMD) Workgroup Leader: Nicole Wolf, MD, PhD (Amsterdam University Medical Centers) Speaker No. 1: Chloe Stutterd, MD, PhD (Murdoch Children’s Research Institute) Speaker No. 2: Gesine Saher, PhD (Max Planck Institute) Speaker No. 3: Jigyasha Sinha, MD (Institute of Neurosciences Kolkata)

9:15 - 10:00 a.m.

Pol III-related Leukodystrophy (4H) Workgroup Leader: Geneviève Bernard, MD, MSc, FRCP (McGill University) Speaker No. 1: Nicole Wolf, MD, PhD (Amsterdam University Medical Centers)

10:00 - 10:15 a.m.

Coffee Break

Session VI 10:15 - 11:00 a.m.

Salla Disease Workgroup Leader: Melissa Wasserstein, MD (Albert Einstein College of Medicine) Speaker No. 1: David Adams, MD, PhD (National Human Genome Research Institute) Speaker No. 2: Marjan Huizing, PhD (National Human Genome Research Institute)

11:00 - 11:45 a.m.

TUBB4A-related Leukodystrophy Workgroup Leader: Adeline Vanderver, MD (Children’s Hospital of Philadelphia) Speaker No. 1: Sunetra Sase, PhD (Children’s Hospital of Philadelphia) Speaker No. 2: Henry Houlden, MD, PhD (University College London)

11:45 a.m. - 12:00 p.m.

Group Photo

12:00 - 1:00 p.m.

Lunch

Session VII

1:00 - 1:45 p.m.

Cerebrotendinous Xanthomatosis (CTX) Workgroup Leader: Andrea DeBarber, PhD (Oregon Health & Science University) Speaker No. 1: Robert Steiner, MD (University of Wisconsin)

1:45 - 2:30 p.m.

Autosomal-Dominant Leukodystrophy (ADLD) Workgroup Leader: Quasar Padiath, MBBS, PhD (University of Pittsburgh) Speaker No. 1: Julia Kofler, MD (University of Pittsburgh) Speaker No. 2: Raili Raininko, MD, PhD (Uppsala University)

2:30 - 2:45 p.m.

Coffee Break

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Session VIII 2:45 - 3:30 p.m.

Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP) Workgroup Leader: Jennifer Orthmann-Murphy, MD, PhD (University of Pennsylvania) Speaker No. 1: Troy Lund, MD, PhD, FAAP (University of Minnesota) Speaker No. 2: Josh Bonkowsky, MD, PhD (University of Utah)

3:30 - 4:15 p.m.

Refsum Disease (Adult) Workgroup Leader: Joseph Hacia, PhD (University of Southern California) Speaker No. 1: Florian Eichler, MD (MassachusettsGeneral Hospital) Speaker No. 2: Kristie DeMarco, BS (Global DARE Foundation)

4:15 - 4:30 p.m.

Closing Remarks and Meeting End

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