4th ANNUAL SCIENTIFIC
SYMPOSIUM ON SPG4

Wednesday, March 25-Friday, March 27
Boston, MA or Virtual
Co-hosted by The Lilly and Blair Foundation and Boston Children’s Hospital

In-person registration is now closed. Virtual registration remains open through Monday, March 23.

Agenda and Assessment Info
Registration
Hotel Information

The premier gathering for childhood-onset SPG4 research returns in 2026 — uniting leading scientists, clinicians, industry innovators, patient advocates, and families for three days of focused collaboration. Designed to accelerate the path from discovery to treatment, the symposium will feature experts from across the U.S. and abroad, offering a rare opportunity to share knowledge, align priorities, and drive progress.

The centerpiece of the event, the Scientific Symposium will take place on Thursday, March 26, bringing researchers together to share the latest advances in gene therapy, drug repurposing, model validation, and near-term therapeutic targets. These discussions will be bookended by SP-CERN clinical assessments and intakes on Wednesday and Friday, significantly expanded from last year to allow for broader participation and more in-depth evaluations.

We also plan to create opportunities for families to connect through informal networking, happy hours, or group activities — making this not only a scientific milestone but a community-building event. Scroll down for more details about this year’s event, view last year’s symposium summary, or learn more about the SP-CERN.

Registration Options

  • In-Person Registration: CLOSED

    Your in-person registration fee covers your entire family attending in person under one order. Registration includes full access to all sessions, networking opportunities with researchers and families, and on-site materials.

    In-person registration is open to all individuals wishing to attend the Symposium in Boston. This includes families of patients with childhood-onset SPG4 who are interested in SP-CERN assessment and enrollment, as well as anyone affiliated with SPG4 who would like to learn more.

    Registration fees help cover essential event costs such as room rentals, audio/visual support, and materials needed to bring this important gathering to life.

  • Virtual Registration: $25

    Your $25 registration includes live-stream access to all sessions, digital conference materials, and the chance to submit questions during Q&A sessions with researchers and speakers.

    Registration fees help cover essential event costs, including room rentals, audio/visual support, and the technology that makes virtual participation possible.

    View Details and Register Now

Sponsorship

View sponsorship benefits and confirm your support below or download the pdf for details. In addition to the sponsorship-specific benefits listed, all sponsors receive:

  • Logo or name on the Symposium webpage

  • Listing in pre-event communications and digital agenda

  • Inclusion in the digital program and session descriptions

  • Acknowledgment on opening and closing slides

  • Recognition in the post-Symposium summary

  • Premier visibility as the lead supporter of the 2026 SPG4 Scientific Symposium.

    • Exclusive top-tier logo placement across all Symposium materials

    • Recognition in opening remarks

    • Optional 30–45 second recorded message during a plenary transition

    • Featured placement in the post-Symposium summary

    • Option to include a one-page PDF (“About Our Organization”) in attendee materials

    • Ability to provide branded items/literature in attendee bags

    • Top placement in the sponsor one-pager (quote, logo, statement, link)

    • Logo displayed before one major scientific session

    • Recognition in opening remarks of one Family Day session

    • Ability to provide branded materials for attendee bags

    • Prominent placement in the sponsor one-pager (logo, statement, link)

    • Ability to place company literature in attendee folders

    • Prominent placement in the sponsor one-pager (logo + link)

  • Logo included in the sponsor one-pager (logo + link)

  • Community Sponsors receive all standard sponsor recognition listed above

Confirm Your Sponsorship Now

For organizations seeking broader, year-long impact and visibility, The Lilly and Blair Foundation also offers Corporate Partnership opportunities across all signature platforms. Learn more at lillyandblair.org/partnership.

Agenda at a Glance

This year’s symposium will focus on childhood-onset SPG4 and includes opportunities for families, researchers, and clinicians to connect. The full agenda is still in development. Stay tuned for updates and additional speaker announcements.
Download the draft agenda here.

Wednesday, March 25 | Assessment and Family Day

Assessments: 2 Brookline Place, Brookline, MA 02445  ·  Family Day: 700 Brookline Avenue, Brookline, MA 02446
1:30–1:45 pm
Welcome and Orientation
Darius Ebrahimi-Fakhari, MD, PhD — Boston Children’s Hospital and Harvard Medical School
Katie Gregg — The Lilly and Blair Foundation
Chris Lorek — The Lilly and Blair Foundation
1:45–2:45 pm
Research 101: Therapies and Models That Drive Progress
Miguel Sena-Esteves, PhD — UMass Chan Medical School
Heather Gray Edwards, DVM, PhD — UMass Chan Medical School
Peter Baas, PhD — Drexel University College of Medicine
Liang Oscar Qiang, MD, PhD — Drexel University College of Medicine
2:45–3:00 pm Break
3:00–3:30 pm
Understanding the Research Pipeline
Darius Ebrahimi-Fakhari, MD, PhD — Boston Children’s Hospital and Harvard Medical School
Rotem Or Bach, MD — The National Institutes of Health (NIH)
3:30–4:30 pm
Clinical Trials Demystified
Steven Gray — UT Southwestern*
Michael Shy, MD — University of Iowa
5:30-8:00 pm Networking Evening at Punch Bowl, Hilton

Thursday, March 26 | Scientific Symposium Day

Venue: Karp Family Research Laboratories, Karp Boardroom, 1 Blackfan Street, Boston, MA 02115
9:15–9:45 am Registration
9:45–10:00 am
Welcome and Overview
Darius Ebrahimi-Fakhari, MD, PhD — Boston Children’s Hospital and Harvard Medical School
Katie Gregg — The Lilly and Blair Foundation
Chris Lorek — The Lilly and Blair Foundation
Session 1: Accelerate Pre-Clinical Progress for Gene-Replacement Therapy
10:00–10:20 am
Next Generation Dual-Function AAV Vectors for SPAST Silencing and Replacement
Miguel Sena-Esteves, PhD — UMass Chan Medical School
10:20–10:40 am
Preclinical Evaluation of SPAST Gene Replacement in Novel SPG4 Mouse Models
Emanuela Piermarini, PhD — Drexel University College of Medicine
10:40–11:00 am
Refining the Path to Treatment: AAV-Mediated SPAST Gene Delivery in the SPG4 Rat Model
Molly Lettman, PhD — University of Wisconsin–Madison*
11:00–11:20 am
Defining Pre-IND Pathways for SPAST Gene Therapy: Regulatory and Manufacturing Perspectives
Heather Gray-Edwards, PhD, DVM — UMass Chan Medical School
11:20–11:40 am
Questions and Comments
Moderators:
Stefan Hauser, PhD — German Center for Neurodegenerative Diseases (DZNE)*
Steven Gray, PhD — UT Southwestern*
11:40–11:55 am Coffee Break
Session 2: Validate Models and Integrate Insights
11:55 am–12:15 pm
Probing Disease Mechanisms: Contributions of Isoforms and Catalytic Domain Mutations
Julia Flood — University of Wisconsin–Madison
12:15–12:35 pm
Isogenic Forebrain Organoids Illuminate Genotype-Phenotype Mechanisms in SPG4
Liang Oscar Qiang, MD, PhD — Drexel University College of Medicine
12:35–12:55 pm
Preclinical Mouse Model Development for the SPG4 p.Arg499His Variant
Peter Baas, PhD — Drexel University College of Medicine
12:55–1:15 pm
Questions and Comments
Moderators:
Craig Blackstone, MD, PhD — Massachusetts General Hospital and Harvard Medical School
Stephan Zuchner, MD, PhD — University of Miami
1:15–2:00 pm Lunch Break
Session 3: Accelerate Clinical Trial Readiness
2:00–2:20 pm
Clinical and Molecular Genotype-Phenotype Correlations in Childhood-Onset SPG4
Julian Alecu, MD — Boston Children’s Hospital
2:20–2:40 pm
SP-CERN Multi-Center Natural History, Registry and Biorepository Efforts
Darius Ebrahmi-Fakhari, MD, PhD — Boston Children’s Hospital and Harvard Medical School
Stephan Zuchner, MD, PhD — University of Miami
Joshua Burns, PhD — St. Jude Children’s Research Hospital
Michael Shy, MD — University of Iowa
2:40–3:10 pm
International Collaborative Initiatives Toward Trial Readiness
Rebecca Schüle, MD — Heidelberg University Hospital and TreatHSP*
Marcondes França Jr., MD, PhD — State University of Campinas (UNICAMP)*
Seungbok Lee, MD, PhD — Seoul National University* (recording)
3:10–3:30 pm
Questions and Comments
Moderators:
Matthew Elrick, MD, PhD — Kennedy Krieger Institute and Johns Hopkins School of Medicine
Rotem Or Bach, MD — The National Institutes of Health (NIH)
3:30–3:40 pm Coffee Break
Session 4: Identify and Prioritize Near-Term Potential Therapies
3:40–4:00 pm
HDAC Inhibitors and Small-Molecule Modulators
Liang Oscar Qiang — Drexel University College of Medicine
4:00–4:20 pm
M1-Targeted Antibody Strategy and Biomarker Development
Peter Baas, PhD — Drexel University College of Medicine
4:20–4:40 pm
Questions and Comments
Moderators:
Craig Blackstone, MD — Massachusetts General Hospital and Harvard Medical School
Michael Shy, MD — University of Iowa
4:40–5:00 pm
Wrap up
Darius Ebrahmi-Fakhari, MD, PhD — Boston Children’s Hospital and Harvard Medical School
Katie Gregg — The Lilly and Blair Foundation
Chris Lorek — The Lilly and Blair Foundation
5:30-8:00 pm Networking Evening at Yard House, Fenway/Back Bay

Friday, March 27 | Assessment and Family Day

Assessments: 2 Brookline Place, Brookline, MA 02445  ·  Family Day: 700 Brookline Avenue, Brookline, MA 02446
8:30–9:00 am
Scientific Symposium Recap
Darius Ebrahmi-Fakhari, MD, PhD — Boston Children’s Hospital and Harvard Medical School
9:00–10:00 am
Ask the Experts: Open Q&A Panel
Miguel Sena-Esteves, PhD — UMass Chan Medical School
Darius Ebrahmi-Fakhari, MD, PhD — Boston Children’s Hospital and Harvard Medical School
Matthew Elrick, MD, PhD — Kennedy Krieger Institute and Johns Hopkins School of Medicine
Peter Baas, PhD — Drexel University College of Medicine
Liang Oscar Qiang, MD, PhD — Drexel University College of Medicine
10:00–10:15 am Coffee Break
10:15–10:45 am
Therapeutic Strategies at Home
Elizabeth Hodge, PT, DPT, PCS, CEIS — Boston Children’s Hospital
Julia Shah, MD — Boston Children's Hospital, Spaulding Rehabilitation, and Harvard Medical School
Zainab Zaman, MD — Boston Children’s Hospital
10:45–11:15 am
Family Networking and Resource Sharing
Zona Reid — Parent, Author of 499 Parent Resource Guide and Collaborator on Childhood-Onset HSP Website
Bridget Lassig — Parent, Michigan and Childhood-Onset HSP Survey Contact, Collaborator on Childhood-Onset HSP Website
11:15 am–12:15 pm
Coping, Resilience, and Support
Amanda Griffith-Atkins, MS, LMFT — Therapist and Founder of Amanda Atkins Counseling Group; Author of "How to Handle More Than You Can Handle"