ALD Connect Annual Meetings


Every year, ALD Connect hosts an Annual Meeting that brings all ALD stakeholders together—patients, caregivers, patient advocates, clinician/researchers, and pharmaceutical companies—to present the latest developments in ALD research, clinical trial information, and patient perspectives, and to discuss how we can work together to advance new therapies with the goal of curing ALD.

ALD Connect Annual Meeting 2019

November 8-9, 2019 – Waltham, MA

Videos from the 2019 Annual Meeting

Note: 2019 Patient Learning Academy videos can be viewed here.

Welcome: Myelin Project and ALD Updates  (Florian Eichler)

Big Data & Little Fish (Josh Bonkowsky)

ALD Newborn Screening – Progress and Challenges (Elisa Seeger)

SCAN: Screening for ALD in the Netherlands (Stephan Kemp)

Newborn Bloodspot Screening: A Global Perspective (Bob Wyborn)

GLIA-CTN Global Leukodystrophy Initiative – Clinical Trials Network (Adeline Vanderver)

Ask an Expert: Patient Panel (Ben Lenail)

ALD Connect Awards

Engraftment Disease Remission & Clinical Trial Update (Troy Lund)

Early Lesion Growth Analysis in Cerebral ALD (Erik Mallack)

Pilot Scale Study of Human Plasma Identifies Potential Biomarkers to Predict X ALD (Jaspreet Singh)

Generation of ALD Brain Organoids as a Preclinical Model (Vivi Heine)

Update on Dendrimer TherapyOrganoids as a Preclinical Model (Ali Fatemi)

Biomarker Discovery for Disease Onset or Therapeutic Intervention Studies (Yorrick Jaspers)

Industry Panel (Amber Salzman)

ALD Connect Annual Meeting 2018

November 2nd and 3rd, 2018 – Philadelphia, PA

Videos from the 2018 Annual Meeting

Note: 2018 Patient Learning Academy videos can be viewed here.

Welcome: Updates on ALD and AMN  (Florian Eichler)

Centers of Excellence Standards & Care Management (Adeline Vanverver)

LCN Clinical Practice Guidelines (Anna Grantham)

Newborn Screening Response and Long-Term Monitoring (Laura Adang)

Imaging Advances in AMN: Spinal Cord (Bela Turk)

Modeling Cerebral ALD Lesion Growth (Eric Mallack)

Expert Consensus Guidelines – A Transatlantic Approach (Marc Engelen)

Progress in Gene Therapy for ALD (Christine Duncan)

Risk & Rewards Stabilizing ALD With Transplant (Troy Lund)

Cord Blood Transplantation for Children (Vinod Prasad)

Understanding Dysfunction of the Blood-Brain Barrier in ALD (Patricia Musolino, Alexander Lenail)

Industry Panel (Cleland/Opheris, DeWitt/Deuterx-Poxel, Fortin/bluebird bio, Meya/Minoryx, Cromwell/Sanofi Genzyme)

ALD Connect Meeting 2017

November 17th and 18th, 2017 – Bethesda, MD

Videos from the Annual Meeting

Welcome: Introductions and Goals for 2017 Annual Meeting (Ali Fatemi)

The Therapeutic Landscape of ALD and AMN (Florian Eichler)

Clinical Trial Readiness for Rare Neurological Diseases: FDA and NIH Perspective (Devanand Jillapalli (FDA)  and Jill Morris (NIH)

The ALD Database and the Need for Translational Metabolism in Newborn Screening (Stephan Kemp)

Newborn Screening for ALD (Ann Moser and Elisa Seeger)

2017 ALD Connect Awards (Kailey Walsh)

Industry Panel: Updates on Trial Plans

Outreach and Advocacy for ALD (Jean Kelly)

Learning from Others: Patient Engagement Across Rare Disease (Ronald Bartek and Margie Frazier)

Group Brainstorm and Wish List for 2018 (Ben Lenail)