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
Neurogene
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