07:30

Canada/Eastern

07:30 - 08:30 EST
RI-MUHC Block E Atrium

Registration and Breakfast

Welcome to the 2025 CARELeuko Symposium! Join our team at the registration table to sign in, pick up your name badge, and enjoy a light breakfast. Poster presenters may also set up their posters during this time.

08:30

Canada/Eastern

08:30 - 08:40 EST
Drs Sylvia et Richard Cruess Ampitheatre

Welcome and Opening Remarks

Dr. Geneviève Bernard, MD, MSc, FRCPc (McGill University Health Centre)

08:40

Canada/Eastern

08:40 - 09:05 EST
Drs Sylvia et Richard Cruess Ampitheatre

Clinical Landscape of Leukodystrophies in Canada

Dr. Sunita Venkateswaran, MD, FRCPc (London Health Sciences Centre)

    Keynote Address

09:05

Canada/Eastern

09:05 - 09:30 EST
Drs Sylvia et Richard Cruess Ampitheatre

Hi-Fi Long-read Genome Sequencing for Leukodystrophy

Dr. Isabelle Thiffault, PhD, FACMGG (Children’s Mercy Kansas City)

    Session I : Early and Accurate Diagnosis

09:30

Canada/Eastern

09:30 - 09:55 EST
Drs Sylvia et Richard Cruess Ampitheatre

Diagnostic Odyssey of Adult-onset Leukodystrophies

Justin Simo (Montreal Neurological Institute)

    Session I : Early and Accurate Diagnosis

09:55

Canada/Eastern

09:55 - 10:20 EST
Drs Sylvia et Richard Cruess Ampitheatre

Newborn Screening in Canada for Leukodystrophies

Dr. Neal Sondheimer, MD, PhD (Children’s Hospital of Eastern Ontario)

    Session I : Early and Accurate Diagnosis

10:20

Canada/Eastern

10:20 - 10:35 EST
RI-MUHC Block E Atrium

Coffee Break

Poster presenters may set up their posters if they haven’t already.

10:35

Canada/Eastern

10:35 - 11:00 EST
Drs Sylvia et Richard Cruess Ampitheatre

Immune Phenotypes in Leukodystrophies

Dr. Donald C. Vinh, MD, FRCPc, FACP (McGill University Health Centre)

    Session 2 : Translational Research in Leukodystrophies

11:00

Canada/Eastern

11:00 - 11:25 EST
Drs Sylvia et Richard Cruess Ampitheatre

iPSC Technology to Study Leukodystrophy Pathophysiology

Dr. Thomas Durcan, PhD (Montreal Neurological Institute)

    Session 2 : Translational Research in Leukodystrophies

11:25

Canada/Eastern

11:25 - 11:50 EST
Drs Sylvia et Richard Cruess Ampitheatre

Leveraging RNA-based Therapeutics for ALSP

Dr. Jo Anne Stratton, PhD (Montreal Neurological Institute)

    Session 2 : Translational Research in Leukodystrophies

11:50

Canada/Eastern

11:50 - 12:15 EST
Drs Sylvia et Richard Cruess Ampitheatre

Leveraging ASOs for POLR3-related Leukodystrophy

Samuel Gauthier (McGill University Health Centre Research Institute)

    Session 2 : Translational Research in Leukodystrophies

12:15

Canada/Eastern

12:15 - 12:40 EST
Drs Sylvia et Richard Cruess Ampitheatre

Leveraging Gene Therapy for MLD

Dr. Stephanie Newman, PhD (London Health Sciences Centre)

    Session 2 : Translational Research in Leukodystrophies

12:40

Canada/Eastern

12:40 - 14:10 EST
RI-MUHC Block E Atrium

Lunch and Poster Session

Poster viewing begins at 1:10 PM.

14:10

Canada/Eastern

14:10 - 14:35 EST
Drs Sylvia et Richard Cruess Ampitheatre

Importance of Clinician-Researcher Collaborations

Dr. Laurence Gauqelin, MD (CHU de Québec - Université Laval)

    Session 3 : Clinical Research and Family Impact

14:35

Canada/Eastern

14:35 - 15:00 EST
Drs Sylvia et Richard Cruess Ampitheatre

The Role of Leukodystrophy Advocacy in Canada

Riham Badawy (ALD Hope)

    Session 3 : Clinical Research and Family Impact

15:00

Canada/Eastern

15:00 - 15:30 EST
Drs Sylvia et Richard Cruess Ampitheatre

Patient Representative Q&A

Moderators: Sabrina Alam, PhD and Ruwan Bedeir

    Session 3 : Clinical Research and Family Impact

15:30

Canada/Eastern

15:30 - 15:45 EST
RI-MUHC Block E Atrium

Coffee Break

Poster presenters may also take down their posters at this time or at the end of the Symposium.

15:45

Canada/Eastern

15:45 - 16:10 EST
Drs Sylvia et Richard Cruess Ampitheatre

Canadian Partnerships in Rare Disease Research

Dr. Thierry Lacaze-Masmonteil, MD, PhD (RareKids-CAN)

    Session 4 : Cross-National-Border Collaboration

16:10

Canada/Eastern

16:10 - 16:35 EST
Drs Sylvia et Richard Cruess Ampitheatre

Rare Disease Policy in Canada

Dr. Francois Bernier, MD (University of Calgary)

    Session 4 : Cross-National-Border Collaboration

16:35

Canada/Eastern

16:35 - 17:00 EST
Drs Sylvia et Richard Cruess Ampitheatre

Developing Leukodystrophy Research and Care Infrastructure

Dr. Adeline Vanderver, MD (Children’s Hospital of Philadelphia)

    Session 4 : Cross-National-Border Collaboration

17:00

Canada/Eastern

17:00 - 17:25 EST
Drs Sylvia et Richard Cruess Ampitheatre

Indigenous Health in Research and Care

Representative from Cree Board of Health and Social Services of James Bay

    Session 4 : Cross-National-Border Collaboration

17:25

Canada/Eastern

17:25 - 17:40 EST
Drs Sylvia et Richard Cruess Ampitheatre

Closing Remarks

Dr. Roberta La Piana, MD, PhD (Montreal Neurological Institute)

18:00

Canada/Eastern

18:00 - 21:30 EST
PK Subban Atrium

2025 CARELeuko Symposium - Networking Event

Please join us after the symposium for dinner in the PK Subban Atrium at the Montreal Children’s Hospital. This is the perfect opportunity to connect with the Leukodystrophy community, and continue conversations sparked throughout the day. We hope to see you there!

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