Fifth International Symposium on Myelin Disorders

ALD Summit - Approaching Therapeutic Goals

June, 04 - 05 2021
Friday 04:30 - 08:15 pm / Saturday 04:00 - 07:30 pm

Missed the event?

You are welcome to view the recordings here: Friday, June 4th 2021 | Saturday, June 5th 2021

PROGRAM

Friday, June 4th 2021

16:30 - 16:40 Opening Remarks, Welcome to the Meeting
Joseph Claßen
Leipzig, GER

16:40 - 17:00 Introduction to the Meeting
Wolfgang Köhler
Leipzig, GER

Session I Keynote Lecture
Chair: Wolfgang Köhler

16:50 - 17:30 Therapy Developments in Leukodystrophies
Marjo van der Knaap
Amsterdam, NL

Session II Pathogenetic Aspects in X-ALD
Chairs: Alfonso Barrantes-Freer, Florian Then Bergh

17:30 - 17:45 Concurrent Axon and Myelin Destruction Differentiates X-ALD from Multiple Sclerosis
Caroline Bergner
Leipzig, GER

17:45 - 18:00 Metabolic Re-routing of Very-Long-Chain Fatty Acid Synthesis Impacts Adrenoleukodystrophy
Stephan Kemp
Amsterdam, NL

18:00 - 18:15 New Insights in Cerebral Adrenoleukodystrophy
Josh Bonkowsky
Salt Lake City, USA

18:15 - 18:45 Discussion

18:45 - 19:00 Coffee Break

Session III Therapeutic Options, Part 1
Chairs: Jörn Kühl, Florian Eichler

19:00 - 19:15 The Potential of Histone Deacetylase Inhibitors to Halt the Inflammatory Demyelination in Patients with Cerebral ALD
Johannes Berger
Vienna, A

19:15 - 19:30 Is there a Role for VLCFA Normalization?
Marco Cappa
Roma, I

19:30 - 19:45 Lipid Metabolism Disturbances in X-ALD
Beyond VLCFA
Aurora Pujol
Barcelona, ESP

19:45 - 20:00 Leriglitazone in AMN - Results from a Phase 2/3 Clinical Study
Wolfgang Köhler
Leipzig, GER

20:00 - 20:15 Discussion

Saturday, June 5th 2021

Session IV Monitoring Disease Progression
Chairs: Marjo van der Knaap, Johannes Berger

16:00 - 16:15 Disease Progression in Male and Female AMN - Lessions Learned from Natural History Studies
Irene Huffnagel
Amsterdam, NL

16:15 - 16:30 Measuring Severity of Myelopathy in XALD
Marc Engelen
Amsterdam, NL

16:30 - 16:45 Applying Research to Medical Needs: Neurofilament Light Chain (NfL) Protein as a Biomarker for X-linked ALD
Isabelle Weinhofer
Vienna, A

16:45 - 17:00
Biomarkers in AMN -The World Beyond NfL
Ali Fatemi
Baltimore, USA

17:00 - 17:30 Discussion

17:30 - 17:45 Coffee Break

Session V
Therapeutic Options, Part II
Chairs: Charles Peters, Basil Sharrack

17:45 - 18:00 Allogeneic Hematopoietic Stem Cell Transplantation in Childhood Cerebral Adrenoleukodystrophy
Troy Lund
Minneapolis, USA

18:00 - 18:15 Allogeneic Hematopoietic Stem Cell Transplantation in Adulthood Cerebral ALD - The Leipzig Leukodystrophy Center Experience
Georg Franke
Leipzig, GER

18:15 - 18:30 Gene Therapy in Cerebral ALD
Jörn Kühl
Leipzig, GER

18:30 - 18:45 Gene Therapy in AMN
Florian Eichler
Boston, USA

18:45 - 19:15 Discussion

19:15 - 19:30 Summary of the Meeting and Take-Home-Messages
Wolfgang Köhler
Leipzig, GER

19:30 END OF THE SCIENTIFIC SYMPOSIUM

Please find the full program here.

Scientific Head

Wolfgang Köhler M.D.
Leukodystrophy Center Leipzig
Dpt. of Neurology, University of Leipzig Medical Center

COOPERATION PARTNER

ORGANISATIONAL MANAGEMENT:
Myelin Projekt Deutschland e.V.
Gottfried-Keller-Straße 9
04289 Leipzig
info@myelin.de
www.myelin.de

Sponsors

Minoryx (10.000 €)

SwanBio Therapeutics (5.000 US$)

Vigil Neuroscience (5.000 US$)

Pharmaelle (1.500 €)

bluebird bio (1.000 €)

ELA Deutschland e.V. (1.000 €)

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