#MSDCon2024
Conference Schedule
7:30am
Registration and Breakfast
7:45am
Poster Drop Off Open
8:00am
Opening Remarks
Sarah Cortell Vandersypen
8:10am
Thomas Dierks Award
Sarah Cortell Vandersypen
8:20am
SECTION 1 Introduction – Road to Clinical Trials
Laura Adang
8:30am
Development of a rigorous approach to performing Real-World Evidence-based natural history studies in leukodystrophies
Anjana Sevagamoorthy
8:50am
Natural History Study of Multiple Sulfatase Deficiency: Updates
Laura Adang
9:10am
Biochemical signatures of disease severity in Multiple Sulfatase Deficiency
Rebecca Ahrens-Nicklas
9:30am
The impact of Multiple Sulfatase Deficiency on children & families: caregiver perspectives
Francesco Gavazzi
9:50am
Section Wrap-Up and Q&A
Moderated by Section Chair
10:10am
COFFEE BREAK
11:00am
SECTION 2 Introduction – Clinical Trials
Rebecca Ahrens-Nicklas
11:10am
Efficacy of a scAAV9/SUMF1 viral vector for the treatment of Multiple Sulfatase Deficiency
Steven Gray
11:30am
GLP toxicology findings for AAV9/SUMF1 intrathecal or intracisternal magna administration in WT rats
Steven Gray
11:50am
The Accelerating Medicines Partnerships® (AMP®) Bespoke Gene Therapy Consortium (BGTC) Regulatory Playbook
Brad Garrison
12:10pm
Repurposing of tazarotene– update on preparations for the first clinical trial for MSD
Lars Schlotawa
12:30pm
The REMEDi4ALL drug repurposing consortium
Donald Lo
12:50pm
Section Wrap-Up and Q&A
Moderated by Section Chair
1:10pm
LUNCH
2:30pm
SECTION 3 Introduction – New Discoveries
Lars Schlotawa
2:40pm
Finding starting points for structure-based drug design
Hartmut Niemann
3:00pm
Recent Progress on the Identification of Small Molecule Reactivators of Sulfatases
Matthias Baud
3:20pm
Patient-derived NGN2-induced neurons recapitulate biochemical phenotypes of Multiple Sulfatase Deficiency in vitro
Margaret M. Cassidy
3:40pm
Single vs. dual transgene ex vivo gene therapy for multiple sulfatase deficiency
Vi Pham
4:00pm
Section Wrap-Up and Q&A
Moderated by Section Chair
4:20pm
BREAK
4:40pm
SECTION 4 Introduction – Looking Forward
Lars Schlotawa
4:50pm
Tracking Clinical and Biochemical Changes Post Bone Marrow Transplant in Multiple Sulfatase Deficiency – A 3-Year Follow-Up
Nishitha Pillai
5:10pm
Newborn Screening for Lysosomal Storage Disorders and Beyond (including MSD)
Michael Gelb
5:30pm
MSD Research Strategy Roadmap
Matthew Jarpe
5:50pm
Section Wrap-Up and Q&A
Moderated by Section Chair
6:10pm
BREAK
6:30pm
Posters Session
Poster Presenters
8:00am
Registration and Breakfast
9:00am
Opening Remarks
Sarah Cortell Vandersypen
9:05am
Scientific Day in Review – Summarized Updates
Dr. Rebecca Ahrens-Nicklas / Dr. Laura Adang / Dr. Lars Schlotawa
10:10am
Integrated Health
Dr. Maria Mascarenhas
11:00am
COFFEE BREAK
11:30am
Integrated Health Demo
Dr. Maria Mascarenhas
12:00pm
Orthopedics
Dr. Chrissy Goodbody
1:00pm
LUNCH
2:00pm
Gastroenterology
Dr. Nikki Jaffe
3:00pm
Collaboration is Everything: Newborn Screening Advocacy for Rare Diseases
Lesa Brackbill
3:45pm
Parent Panel – What I Wish I Would Have Known
Holden Duchon / Alan Finglas / Amber Olsen / Stormi Scotting
4:30pm
Remembrance Activity
4:55pm
Closing Remarks
Sarah Cortell Vandersypen
Sponsorship Information
General
Questions
Media / Press Opportunities
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