ML4 Family and Researcher Conference May 10-11, 2020
Pre-conference NeuroClinic May 8-9
Boston, Massachusetts, USA
Wyndham Boston Beacon Hill Hotel

This biennial meeting begins with a family day of learning, sharing, and support. Leading researchers will join families to explain their work, and to share what they are learning about ML4. They will also be attending to listen and learn from ML4 families and children.

Schedule Overview

Friday, May 8th and Saturday, May 9th

On these two days prior to the conference, we are teaming up with our colleagues at Massachusetts General Hospital (MGH) to hold a neurology and rehabilitative medicine clinic. We want you and your child there to engage with Dr. Albert Misko and Dr. Brian Wishart! Travel stipends are available for participating families.

See more information about NeuroClinic here.

Sunday, May 10th

The first day of the conference features sessions focused on gene therapy as treatment for MLIV including: discussion of research at MGH developing treatment; a presentation from a family whose child has been treated by gene therapy for another rare disease; and information about the process toward a clinical trial. The focus on this day will be to provide a great deal of information to families about all aspects of gene therapy, clinical trials, risks and opportunities of this possible treatment.

Monday, May 11th

The second day includes scientific sessions where leading MLIV researchers will share gene therapy research with leading gene therapy scientists to gain insight and guidance on further developing this treatment for patients. Families are invited to attend.

Schedule details will be posted as soon as they are available.

Register for the Conference

  • 1) Reserve your room at the Wyndham Boston Beacon Hotel

    • Use this link to reserve online:
    • Call (877) 999-3223 and refer to the “ML4 Family and Researcher Conference and/or ID #: 0510ML4CONF”

    These rooms are available at a special rate of $269/night for a limited time, so please book quickly to assure this pricing.

  • 2) Review information about the ML4 NeuroClinic

    If you plan to attend the conference with your ML4 child,  we want you and your child to engage with Dr. Albert Misko and Dr. Brian Wishart at the 2020 NeuroClinic! Participants are eligible to receive a stipend to help cover travel expenses.

    See details about the NeuroClinic here.

For questions or more information about the conference, please contact Executive Director, Rebecca Oberman at

See photos from the 2018 Family and Research Conference here!