Tuesday, July 20, 2021

Update on Newborn Screening Algorithms for Krabbe Disease
Presented by Dr. Dietrich Matern

State Newborn Screening Programs Updates
Kentucky – Dr. Sainan Wei
New York – Dr. Joseph Orsini
Missouri – Tracy Klug
Ohio – Dr. Margie Ream
Tennessee – Dr. Christine Dorley
Illinois – Dr. Khaja Basheeruddin
New Jersey – Caitlin Russo
Indiana – Allison Forkner
Pennsylvania – P.J. Borandi
Georgia – Dr. Patricia Hall

Krabbe NBS Patient Perspective – Tygh’s Story
Presenter: Amanda DeRossett

Leukodystrophy Newborn Screening (NBS) Updates

Krabbe RUSP (Recommended Uniform Screening Panel) Update
Presented by: Dr. Joseph Orsini

Krabbe NBS Council
Presented by: Dr. Joanne Kurtzberg

Krabbe NBS Guidelines
Presented by: Dr. Robert Thompson Stone

IKD NBS Outcomes and Neuroimaging Standardization for Krabbe Disease
Presented by: Dr. Joanne Kurtzberg and Dr. Margie Ream

Updates in ALD NBS
Presented by: Dr. Eric Mallack

Update on MLD NBS
Presented by: Dr. Michael Gelb

Wednesday, July 21, 2021

Clinical Trials and New Treatments for Leukodystrophies
Travere Therapeutics
Orchard Therapeutics
Ionis Pharmaceuticals
Forge Biologics
Passage Bio
Affinia Therapeutics
Gain Therapeutics

Leukodystrophy Care Network (LCN) Care Coordinators – Transition of Care for Children with Leukodystrophy
Presented by: Catherine Becker

LCN Certified Centers Presentations
Kennedy Krieger Institute
Duke Children’s Hospital
Massachusetts General Hospital
Golisano Children’s Hospital
Lurie’s Children’s Hospital
Primary Children’s Hospital
Children’s Healthcare of Atlanta
Nationwide Children’s Hospital
UC Davis Health System
Weill Cornell Medicine

Thursday, July 22, 2021

Gene Therapy 101
Presented by: Dr. Florian Eichler

Clinical Trials 101
Presented by: Dr. Joshua Bonkowsky

Leukodystrophy Care Network Overview
Presented by: Catie Becker, Kim Hollandsworth, Rachel Hickey

Advocacy for Krabbe Newborn Screening – A Family Perspective
Presented by: Bruce Kirbo