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“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

Speakers, Moderators and Group Leaders

MDA gratefully acknowledges the following for their support

Genzyme General
Wyeth Pharmaceutical
Pfizer
PTC Therapeutics
Amicus Therapeutics
CepTor Corporation