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!

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MEET OUR PANELISTS

Dr. Laura Adang

Children’s Hospital of Philadelphia

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Dr. Albert Freedman

Freedman Counseling Associates, West Chester, PA

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Dr. Melissa Wasserstein

 Montefiore Medical Center

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Shanna Quimby

Cure MLD / Gavin Flying for a Cure

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Dr. Bobby Gaspar

Orchard Therapeutics

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Dr. Albert Seymour

Homology Medicines

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Melanie Rumbel

Cure MLD

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Lesa Brackbill

Leukodystrophy Newborn Screening Action Network

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Erica Barnes

(Moderator) Chloe’s Fight Rare Disease Foundation

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Amy Gaviglio

Consultant and Genetic Counselor

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Dr. Jaap-Jan Boelens

(Moderator) Memorial Sloan Kettering Cancer Center

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Dr. David Whiteman

 Takeda Pharmaceuticals

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Dr. Paul Orchard

(Moderator) University of Minnesota

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.  

You are not alone, please contact us and we will have a family ambassador contact you.

We will also send you and your loved one a special care package that includes Loie’s Disease, a children’s book to explain leukodystrophy to children, and the “Living with Leukodystrophy” medical resource guide.

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