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2022 Global Leukodystrophy Initiative Scientific Meeting

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Date:
Oct 10, 2022 ‐ 8:30 am
End Date/Time-NEW
Where:
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The Leukodystrophy Center at Children's Hospital of Philadelphia, in partnership with colleagues throughout the Global Leukodystrophy Initiative (GLIA), will host a two-day scientific meeting October 10-11, 2022.

The meeting is designed to showcase recent clinical and research advances in a group of 17 leukodystrophies poised for significant therapeutic development in the coming years.

The goal will be to to identify opportunities for meaningful new interdisciplinary partnerships to address persistent challenges in the diagnosis, care, and treatment of affected individuals.

Watch Online

Visit https://theglia.org/2022gliaconference to watch the conference.

Monday, October 10

(8:00 a.m. - 5:00 p.m.)

Welcome and Introductions (8:30 - 9:15 a.m.)

  • 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)

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 - 12:00 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)

Session III

1:00 - 1:45 p.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)

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)

Tuesday, October 11

(8:00 a.m. - 4:30 p.m.)

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)

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: Francesco Gavazzi, MD, PhD (Children's Hospital of Philadelphia)
  • Speaker No. 2: Henry Houlden, MD, PhD (University College London)

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)

2:45 - 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