10:00 AM – 10:25 AM
Welcome: Updates on ALD and AMN
Dr. Florian Eichler, Massachusetts General Hospital
Annual Meeting
November 13, 2020 – 10 AM EST to 4:45 PM EST
10:30 AM – 10:55 AM
Non-BMT Resolution of Cerebral ALD
Dr. Troy Lund, University of Minnesota
11:00 AM – 11:25 AM
Results of a Pilot Study of Vitamin D in ALD Boys
Dr. Keith Van Haren, Stanford University
11:30 AM – 11:55 AM
New Insights into Cerebral ALD
Dr. Joshua Bonkowsky, University of Utah
12:00 PM – 12:25 PM
ALD Newborn Screening Pilot Project
Dr. Eric Mallack, Weill Cornell Medicine
12:30 PM – 12:55 PM
Metabolic Rerouting of Very-Long-Chain Fatty Acid Synthesis Impacts Adrenoleukodystrophy
Dr. Stephan Kemp, Amsterdam University Medical Centers
1:00 PM – 1:40 PM
Patient Panel
Moderator: Tim Maguire, Patients: Ken Dieffenbach, Jillian Smith, Sharon Mau, Barbara Machado
1:45 PM – 2:40 PM
Industry Panel
Moderator: Ben Lenail, Presenters: bluebird bio, Inc., ImStem Biotechnology, Autobahn Therapeutics, Minoryx Therapeutics, Poxel, SwanBio Therapeutics, Viking Therapeutics
2:45 PM -3:10 PM
Young Investigator Panel
Moderator: Dr. Ali Fatemi
Dr. Jonanlis Ramirez Alcantara, Massachusetts General Hospital
Dr. Isha Srivastava, Lucile Packard Children’s Hospital
Dr. Ashish Gupta, University of Minnesota
3:15 PM – 3:30 PM
Multi-Omic Approach to Identify Phenotypic Modifiers Underlying Cerebral Demyelination in X-Linked Adrenoleukodystrophy
Phillip Richmond, University of British Columbia
3:30 PM-3:45 PM
Therapeutic Efficacy Potential of PXL770 – A Novel Direct AMPK-Activator and PXL065 – Deuterium-Stabilized R-Pioglitazone – for X-Linked Adrenoleukodystrophy
Dr. Jaspreet Singh, Henry Ford Hospital
3:45 PM – 4:10 PM
Gene Correction in Hematopoietic Stem Cells Restores White Matter Microvascular Function in Cerebral Adrenoleukodystrophy
Dr. Patricia Musolino, Massachusetts General Hospital
4:30 PM – 4:45 PM
ALD Connect Awards
Presented by Kathleen O-Sullivan-Fortin and Greg Benton
Patient Learning Academy
November 14, 2020 – 10 AM EST to 6:00 PM EST
10:00-10:25 AM
Welcome and Introduction to ALD Connect
Kathleen O’Sullivan-Fortin
10:30-10:55 AM
ALD Family Weekend: Building Community
Nic Adler
11:00-11:40 AM
More Than a Diagnosis: Living with ALD and the Mental Health Impact
Johanna Bond, LMHC
11:45-12:30 AM
Breakout Sessions
Community Call Facilitators
12:30-1:15 PM
Endocrine Considerations in Adrenoleukodystrophy
Dr. Molly Regelmann
1:15-1:40 PM (Option 1)
Genetic Counseling
Moderator: Felicity Emerson
Deirdre Sumski, CGC
Janis Sherwood
1:15-1:40 PM (Option 2)
Functional Considerations in Symptomatic Myelopathy Associated with ALD
Dr. Brian Wishart
1:15-1:40 PM (Option 3)
Women with ALD: Current Research, Prevention of Symptoms, Therapies for Symptoms
Dr. Nutan Sharma
Dr. John Winkelman
1:45-2:25 PM (Option 1)
Education Supports for ALD Boys: IEPs and 504 Plans
Moderator: Kathleen O’Sullivan-Fortin
Nicole Metsisto Mazer, Esq.
Bill and Kate Groel
1:45-2:25 PM (Option 2)
Post-Transplant
Moderator: Dr. Troy Lund
Julie Purschke
1:45-2:25 PM (Option 3)
Planning for the Future: Financial Planning
Alexandria Nadworny, CFP, CTFA
Jesse Torrey
2:30 PM-2:55 PM (Option 1)
Grassroots Fundraising
Chris and Katie Marino, X out ALD
2:30 PM-2:55 PM (Option 2)
Multidisciplinary Care and ALD
Catherine Becker, RN/NP
3:00-3:55 PM
Breakout Sessions
Community Call Facilitators
4:00-4:55 PM
Ask the Experts
Dr. Florian Eichler, Dr. Keith van Haren, Dr. Joshua Bonkowsky, Dr. Troy Lund, Dr. Molly Regelmann
5:00-6:00 PM
Social Hour
ALD Connect: 2 Truths and a Lie
Hosted by Jon Kuderer