Children’s Program
Registration opens  12:30 – 2:00

Thursday July 25th

2:00pm

2:10pm

Welcome and Introduction

ISMRD Memorial

Richard Steet/Jackie James

Jackie James/Jenny Noble

Session 1

2:30pm

3:00pm

3:30pm

4:00pm

4:30pm

5:00pm

6:00pm

Key Note Presentation:

TBA

The lysosome in nutrient sensing and cellular grown control

TBA

TAB

Close of Day

Welcome Reception, with Cash Bar and Poster Viewing Session 

Thomas Braulke – Germany

Marco Sardiello –

Roberto Zoncu – 

TBA

TBA

Friday July 26th

Session 2

8:30am

8:55am

9:20am

TBA

Organelle quality control pathways in Niemann-Pick type C disease

TBA

Stuart Kornfeld – USA

Andrew Liebermann – USA

TBA

9:45am

NEU1-mediated desialylation of the MUC1-ectodomain releases a decoy receptor that protects against P.aeruginosa lung infection

Simeon Goldblum – USA

10:15am

Morning Break

Session 3

10:45am

11:10am

11:40am

12:00pm

12:30 -1:30pm

TBA

TBA

Post Doc x2 Abstracts

TBA

Lunch

David Everman – USA

Alessandra d’Azzo – USA

TBA

TBA

Session 4

1:45pm

2:10pm

Preclinical data from canine and feline models of rare genetic diseases

Bone homeostasis and pathology in ML II and ML III

Charles Vite – USA

Sandra Pohl – Germany

2:35pm

Glycosaminoglycans as regulators of cathepsin proteases in lysosomal diseases

Heather Flanagan-Steet – USA

2:50pm

3:15pm

4:10pm

4:30pm

5:30pm

TBA

Afternoon Break

Family Presentation

2x Post doc presentations

End of day – Evening on your own

TBA

Ida Annunziata – USA

TBA

TBA

Saturday July 27th

Session 5

8L30am

8:55am

9:20am

9:45am

10:15am

10:45am

11:10am

12:00pm

12:30pm – 1:30pm

Preclinical studies supporting an AGU gene therapy Trial

Hematopoietic stem cell transplant for the glycoproteinoses

Personalized therapy approaches for Aspartylglucosaminuria

Gene therapy appraches for ML II

Morning Break

Therapy approaches for Fucosidosis in Mice

TBA

2x Post Doc presentations

Lunch

Steven Gray – USA

Troy Lunch – USA

Ritva Tikkanen – Germany

Allison Bradbury – USA

Arne Linorst – Germany

Tim Wood – USA

TBA

Session 6 – Clinical Management, Care and support

1:30pm

2:00pm

2:20pm

2:45pm

3:05pm

3:35pm

4:00pm

4:20pm

5:00pm

6:00 – 7:00pm

7:00 – 9:30pm

Spinal complications – What are they, how to treat

Family Presentation

Pain management and Palliative Care

Family Presentation

Cardiac Issues – What to look out for and how to treat

ERT development for Alpha Mannosidosis – Where are we at?

TBA

TBA

End of Day

Pre Dinner Drinks Cash Bar

Awards Dinner and ISMRD Celebration

TBA

TBA

TBA

TBA

TBA

John Forman – New Zealand

TBA

TBA