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Meeting Slides

"Challenges in Drug Development for Muscle Disease: A Stakeholders' Meeting"
August 4-5, 2005

Original Meeting Brochure (PDF)

Hyatt Regency Bethesda
One Bethesda Metro Center
Bethesda, MD 20814

Co-Chairs:
Jerry Mendell, M.D., Columbus Children's Research Institute
R. Rodney Howell, M.D., University of Miami

Planning Committee
Purpose

THURSDAY, AUGUST 4

8:00 a.m. - 8:30 a.m.	CONTINENTAL BREAKFAST-Diplomat Ambassador Room
8:30 a.m. - 8:35 a.m.	Welcome on Behalf of MDA and Announcements R. Rodney Howell, University of Miami, and MDA Board Member and Scientific Advisory Committee Chair
8:35 a.m. - 8:40 a.m.	The Charge: Main Meeting Goals Jerry Mendell, Children's Research Institute - Slides

Introduction to Drug Development

8:40 a.m. - 8:45 a.m.	Introduction by Moderator Langdon Miller, PTC Therapeutics - Slides
8:45 a.m. - 9:15 a.m.	From "Proof-of-Principle" Trials Through Phase III: The Process Wilson Bryan, FDA - Slides
9:15 a.m. - 9:45 a.m.	FDA Perspective: General Philosophy of Drug Development With a Particular Focus on Muscle Disease Marc Walton, FDA - Slides
9:45 a.m. - 10:15 a.m.	Industry Perspective: Needs for Drug Development A. Philosophy of Drug Development from Industry Perspective Edward Kaye, Genzyme - Slides B. Needs of Industry for Drug Development for Muscle Disease Cristina Csimma, Wyeth - Slides
10:15 a.m. - 10:30 a.m.	COFFEE BREAK

Taking Stock of Natural History Data in Muscle Disease:
Good, Bad and Missing:

10:30 a.m. - 11:00 a.m.	Introduction to Natural History Data in Clinical Trial Design Moderator, Wilson Bryan, FDA - Slides
11:00 a.m. - 11:30 a.m.	Natural History of Pompe Disease Priya Kishnani, Duke University
11:30 a.m. - 12:00 p.m.	Natural History of Duchenne Muscular Dystrophy Phillip Miller, Washington University
12:00 p.m. - 1:00 p.m.	BUFFET LUNCH - Concourse Terrace
1:00 p.m. - 2:00 p.m.	Round Table Review: Natural History of Myotonic Dystrophy, FSHD, LGMD Richard Moxley, University of Rochester - Slides John Kissel, Ohio State University - Slides Kate Bushby, University of Newcastle - Slides
2:00 p.m. - 3:00 p.m.	Break-Out Groups on Natural History: Pompe Disease: Led by Priya Kishnani, Duke University Duchenne Muscular Dystrophy: Led by Craig McDonald, UC Davis Limb-girdle MD: Led by Katie Bushby, University of Newcastle Myotonic Dystrophy: Led by Richard Moxley, University of Rochester FSH Dystrophy: Led by John Kissel, Ohio State University
3:00 p.m. - 3:15 p.m.	COFFEE BREAK
3:15 p.m. - 3:55 p.m.	Group Discussion on Natural History

Challenges in Translational Research

3:55 p.m. - 4:05 p.m.	Introduction to Challenges in Translational Research Moderator, Sharon Hesterlee, MDA - Slides
4:05 p.m. - 4:25 p.m.	NIH on Funding Clinical Trials Scott Janis, NIH Clinical Trials Group - Slides
4:25 p.m. - 4:40 p.m.	FDA Office of Orphan Products Development Grant Program *Sarah Linde-Feucht, FDA - Slides
4:40 p.m. - 5:00 p.m.	Industry Perspective on Translational Research Frank Walsh, Wyeth Pharmaceutical - Slides
5:00 p.m. - 5:30 p.m.	Case Studies and Round Table Discussion Jerry Mendell, Columbus Children's Research Institute: DMD gene therapy; Barry Byrne, University of Florida: Pompe gene therapy - Slides Katie Bushby, University of Newcastle: Exon-skipping for DMD - Slides
6:00 p.m -	DINNER-Diplomat Ambassador Room

FRIDAY, AUGUST 5

8:00 a.m. - 8:30 a.m.

BREAKFAST-Diplomat Ambassador Room

Clinically Significant Endpoints for Muscle Disease Trials

8:30 a.m. - 8:45 a.m.	Introduction to Clinically Meaningful Endpoints for Muscle Disease Moderators, Jane Scott, FDA and Marc Walton, FDA
8:45 a.m. - 9:15 a.m.	Muscle Strength Testing Diana Escolar, Children's National Medical Center - Slides
9:15 a.m. - 9:45 a.m.	Functional Assessments Craig McDonald, UC Davis
9:45 a.m. - 10:15 a.m.	Patient Reported Outcomes Ted Abresch, UC Davis - Slides Jane Scott, FDA - Slides
10:15 a.m. - 10:30 a.m.	COFFEE BREAK
10:30 a.m. - 11:00 a.m.	Pulmonary and Cardiac Assessments Hugh Allen, Columbus Children's Hospital - Slides Jonathan Finder, University of Pittsburgh - Slides
11:00 a.m. - 11:30 a.m.	Surrogate Markers: Enzyme Levels, Muscle Histology and Gene Expression Steve Moore, University of Iowa - Slides
11:30 a.m. - 12:00 p.m.	Muscle Imaging Glenn Walter, University of Florida - Slides
12:00 p.m. - 1:00 p.m.	BUFFET LUNCH-Concourse Terrace
1:00 p.m. - 1:15 p.m.	Safety Endpoints Langdon Miller, PTC Therapeutics - Slides
1:15 p.m. - 2:15 p.m.	Breakout Groups Muscle Strength Testing: Led by Diana Escolar, Children's National Medical Center Functional Testing: Led by Craig McDonald, University of Iowa Patient Assessed Outcomes: Led by Ted Abresch, UC Davis Pulmonary and Cardiac Endpoints: Led by Jonathan Finder, University of Pittsburgh Surrogate Markers: Led by Steve Moore, University of Iowa Muscle Imaging: Led by Glenn Walter, University of Florida
2:15 p.m. - 3:00 p.m.	Group Discussion on Endpoints Moderators, Jane Scott, FDA and Marc Walton, FDA

Summary and Wrap-Up

3:00 p.m. - 3:20 p.m.	Lessons Learned and the Way Forward Jerry Mendell, Columbus Children's Research Institute
3:20 p.m. - 3:50 p.m.	Final Round Table Discussion with Moderators from All Main Sessions
3:50 p.m. - 4:00 p.m.	Conclusions R. Rodney Howell, University of Miami