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Recent MDCC Meeting

April 30, 2018

Neuroscience Center
Conference Room C/D
6001 Executive Blvd
Bethesda, Maryland 20892

Monday, April 30, 2018

Neuroscience Center
Conference Room C/D
6001 Executive Blvd
Bethesda, Maryland 20892

Meeting Agenda

Welcome and introductions

8:30 – 8:45

Walter Koroshetz, MDCC Chair

NIH Report

8:45 – 9:05

Walter Koroshetz, NINDS

Conflict of interest policies and general meeting information

9:05 – 9:15 

Glen Nuckolls, MDCC Executive Secretary

Presentations by New MDCC Patient Organization (ad hoc) Members

9:15-9:30

Grace Pavlath, Muscular Dystrophy Association of America

9:30-9:45 

Gustavo Dziewczapolski, Cure CMD (congenital muscular dystrophy)

9:45-10:00

Ryan Fischer, Parent Project Muscular Dystrophy

Break

10:00-10:15

Presentations by New MDCC Federal Agency (ad hoc) Members

10:15-10:30

Kimberly Richey, Department of Education

10:30-10:45

Eugene Freund, Centers for Medicare and Medicaid Services

10:45-11:00

Catharine Riley, Health Resources and Services Administration

11:00-11:15

Natalie Street, Centers of Disease Control and Prevention

Meeting Reports

11:15-11:30

Muscular Dystrophy Association Clinical Conference
Grace Pavlath, MDA

11:30-11:45

National Duchenne Clinician Conference – PPMD
Ryan Fischer, PPMD

Senator Paul Wellstone Muscular Dystrophy Research Centers Program

11:45-12:00

Assessment of the Wellstone Centers Program
Heather Rieff, NINDS

Lunch

12:00-12:45

Lessons Learned from Drug Approvals in Rare Neuromuscular Diseases

12:45-1:00

Session Overview and Goals
Kenneth H. Fischbeck, NINDS

1:00-1:30

Facilitating SMA patient access to new treatments
Kenneth Hobby, Cure SMA

1:30-2:00

Facilitating DMD patient access to new treatments
Annie Kennedy, Parent Project Muscular Dystrophy

2:00-2:30

Lessons learned from the industry perspective
Edward M. Kaye, Stoke Therapeutics

2:30-3:00

Lessons learned by physicians and professional societies
Nicholas Johnson, University of Utah

3:00-3:30

Facilitating access to treatments through Medicare, Medicaid and CHIP
Eugene Freund, Center for Medicare and Medicaid Services

MDCC group discussion of lessons learned from recent drug approvals

3:30-4:15

Considering strategies for all dystrophies to promote access to new drugs and facilitate integration of new treatments into the standards of care.

Closing remarks

4:15-4:30

Walter Koroshetz, NINDS

Adjourn

4:30

Meeting Summary

The summary will be posted after the meeting has concluded.