Muscular Dystrophy Coordinating Committee Meeting | Muscular Dystrophy Coordinating Committee

Friday, December 08, 2023

December 8, 2023
Hybrid | 9- 4pm EST

Muscular Dystrophy Coordinating Committee Meeting - 12/8/2023
Time	Presentation
9:00 – 9:15	Introductions and Meeting Information Glen Nuckolls, National Institute of Neurological Disorders and Stroke (NINDS/NIH), MDCC DFO
9:15 – 9:35	Chair Presentation Diana Bianchi, Director, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD/NIH), MDCC Chair
9:35 – 9:45	Group Discussion
9:45 – 10:05	New member nominee presentation Radha Holavanahalli, National Institute on Disability, Independent Living, and Rehabilitation Research (NIDILRR), Administration for Community Living (ACL)
10:05 – 10:50	Meeting/Conference Reports NINDS Digital Endpoints Workshop Carol Taylor-Burds, NINDS/NIH FSHD Society International Research Congress TBD, FSHD Society 2023 Myotonic Dystrophy Foundation Annual Conference Andy Rohrwasser, Myotonic Dystrophy Foundation
10:50 – 11:00	Break
11:00 – 12:00	Meetings/Conference Reports (continued) AAV tox workshop update Carsten Bönnemann, NINDS/NIH Myotonic Dystrophy Young Investigator Conference Andy Berglund, University of Albany Limb-Girdle Muscular Dystrophy International Conference Brad Williams, Jain Foundation and Kathryn Bryant, The Speak Foundation Parent Project Muscular Dystrophy Annual Conference and Specialty Meetings Eric Camino, Parent Project Muscular Dystrophy
12:00 - 12:40	Recent Advances in Duchenne Muscular Dystrophy Session Introduction Glen Nuckolls, NINDS/NIH DMD Gene Therapy Trial Experience Jennifer Handt, Charlie’s Cure and Lianna Orlando, CureDuchenne Group discussion
12:40 – 1:15	Lunch break
1:15 – 2:35	Recent Advances in Duchenne Muscular Dystrophy (session continued) Progress on Newborn Screening for DMD Niki Armstrong, Parent Project Muscular Dystrophy Clinical Trial Simulation Tool for DMD Bill Rooney, OHSU School of Medicine C-Path's Duchenne Regulatory Science Consortium: Data-Driven Solutions to Enhance Drug Development Ramona Belfiore-Oshan, Critical Path Institute Session Summary Glen Nuckolls, NINDS/NIH Group Discussion
2:35 - 2:45	Break
2:45 – 3:30	Advocacy Group Strategic Plans (Continuation from the Spring 2023 MDCC meeting) Muscular Dystrophy Association Angela Lek, Muscular Dystrophy Association Parent Project Muscular Dystrophy Eric Camino, Parent Project Muscular Dystrophy
3:30 – 3:45	Update on Renewal of the MDCC Action Plan Glen Nuckolls, NINDS/NIH Group Discussion on Strategic Planning
3:45 – 4:00	Closing remarks Diana Bianchi, NICHD/NIH
4:00	Adjourn