MLD Standards of Care
Through this event series, we hope to provide meaningful updates about MLD across several themes: advocacy, newborn screening, and clinical research.
EVENT DATES & TIMING
Friday, February 26th: Session 1 | MLD Advocacy & What We’ve Learned
Friday, March 5th: Session 2 | MLD Newborn Screening: ScreenPlus, RUSP and Next Steps
Friday, March 12th: Session 3 | Updates in MLD Clinical Therapies
10:00am – 12:00pm EST
10:00am – 10:45am: Intro & Presentations
10:45am – 11:30am: Moderated Discussion with structured questions
11:30am – 12:00pm: Q&A and Networking
Cure MLD is pleased to present our 2021 MLD Standards of Care virtual conference. While we regret that we were unable to hold this conference in person because of COVID-19, we extend our best wishes to each and every one of you. Through this event series, we hope to provide meaningful updates across several themes: advocacy, newborn screening, and clinical research. We hope you feel informed, hopeful, and even inspired to continue the amazing work this community has completed. Thank you for your interest and collaboration!
MEET OUR PANELISTS
Session 1| MLD Advocacy & What We’ve Learned
February 26th, 10:00am - 12:00pm EST
This mix of speakers from different areas of the leukodystrophy and rare disease community will provide updates from their respective spaces and share what they’ve learned. Speakers will present for 10 minutes each, followed by 45 minutes of moderated discussion. The remaining time will be used to answer questions from the audience. This panel spans several themes including: use of an MLD patient contact registry, updates from CHOP’s MLD Natural History Study, newborn screening advocacy, and providing psychological support for families.
Session 2 | MLD Newborn Screening: ScreenPlus, RUSP and Next Steps
March 5th, 10:00am - 12:00pm EST
These panelists working hard on newborn screening (NBS) will provide updates on MLD NBS from different perspectives. The three speakers will present for 10 minutes each with an hour for moderated discussion. The discussion will be used to direct collaboration to advance agreed upon action items, and will include a suggested timeline and concrete next steps to coordinate work among advocates, clinicians, and researchers. The remaining time will be used to answer questions from the audience.
Session 3 | Updates in MLD Clinical Therapies
March 12th, 10:00am - 12:00pm EST
This final panel will allow industry leaders to interface with the MLD community and provide updates about their clinical therapies. Each industry representative will have 10 minutes to present an update about the status of their therapies. The next hour will be used for moderated discussion about what these updates mean for MLD patients and the community, with a focus on gene therapy. The remaining time will be used to answer questions from attendees. Topics covered will include Orchard’s gene therapy (EMA approval, FDA status, compassionate use), Takeda’s enzyme replacement therapy (Embolden updates), and Homology’s gene therapy development.