Parent Project Muscular Dystrophy (PPMD) fights to end Duchenne Muscular Dystrophy. We accelerate research, raise our voices to impact policy, demand optimal care for every single family, and strive to ensure access to approved therapies.

I will be attending the annual PPMD Conference this Sunday March 3, 2019 through next Tuesday March 5, 2019. Here is the Agenda…

2019 Advocacy Conference

3 Day Agenda


Advocate Training

Mayflower Hotel, 1127 Connecticut Ave, NW, Washington, DC 20036

Room: State Room

2:15pm Registration opens

3:00 – 3:15 Welcome Address & Review of Conference Activities

Pat Furlong, Annie Kennedy, Ryan Fischer

3:15 – 3:35 Congressional Environment – What are we walking into

Nick Manetto, Faegre BD

3:35 – 4:00 Perspective from a former House member

Representative Luke Messer

4:00 – 4:40 Duchenne Advocacy – Making an Impact

Annie Kennedy and Ryan Fischer

4:40 – 4:50 Why Advocacy Matters

Joel Wood

4:50 – 5:30 Advocacy Agenda – This year’s “ask”

Preparing for your meetings

Annie Kennedy & Ryan Fischer

5:30 – 6:00 Soapbox Consulting – Meeting schedules

6:00 – 7:00 Dinner served and break into state groups


Duchenne Gene Therapy Policy Forum

Mayflower Hotel – State Room ¨ 9:00am – 3:30pm

8:15am Registration opens (Breakfast served 8am)

9:00 – 9:10 Welcome and Overview of Forum

Abby Bronson, SVP Research Strategies, PPMD

Session 1 – Where We Are Today

9:10 – 9:40 CBER Update: Agency update on new guidances and where we are since 2016

Celia Witten, MD, PhD, CBER

9:40 – 10:25 Presentations by Companies (15 minutes each)

Pfizer, Sarepta, Solid

Each company will provide update of current gene therapy program

10:25 – 10:40 Q&A from Audience and Discussion with Three Companies

(Moderated by Tim Franson, MD, YourEncore)

Session 2 – AAV in Gene Therapy

10:40 – 11:00 AAV in Duchenne

Lee Sweeney, PhD, Paul Wellstone MD Cooperative Center (UF)

The use AAV in Gene Therapy, what we know from animal models, and work done leading up to human trials.

11:00 – 11:20 Overview of AAV and Use of AAV in Other Diseases

Tim Cripe, MD, Nationwide Children’s Hospital

Overview of what we know in humans affected by other diseases

Session 3 – Gene Therapy Hurdles

11:20 – 11:40 Titers, Testing, and Screening

Barry Byrne, MD, University of Florida

A discussion about prescreening for antibodies and current questions community is raising around screening and titer testing. What are the implications for getting a private test done?

Shouldn’t families know if they are “out of the game” so they can make other choices?

11:40 – 12:20 Getting Around AAV Issues in Gene Therapy and the Potential for Redosing.

(Moderated by Abby Bronson, SVP Research Strategies, PPMD)

Panelists: Tim Cripe, MD, Nationwide Childrens; Joe Kornegay Texas A&M;

Dongshen Duan, PhD, University of Missouri; Olivier Danos, PhD, REGENXBIO

Panel Topics include:

• What are ‘neutralizing antibodies’?

• Those who test positive during screening – what next?

• Plasmaphereses – level setting on where this is in development and if it is a plausible next step strategy in Duchenne gene therapy

• Preparing for screening

• How/can we get to re-dosing?

12:20 – 12:40 Audience Q&A and discussion

(Moderated by Abby Bronson, SVP Research Strategies, PPMD)

12:40 – 1:30 Lunch

Session 4 – The Patient Voice

1:30 – 1:55 Results of PPMD Preference Studies on GT

Ryan Fischer, SVP Community Engagement, PPMD

1:55 – 2:40 Patient Voice Panel – panel of families discussing GT

Moderated by Pat Furlong, Founding President & CEO, PPMD

Session 5 – Looking Toward Future Strategies in Gene Therapy

2:40 – 3:00 What We are Learning About Immunology and the Future for GT

Carrie Miceli, PhD, UCLA

3:00 – 3:15 Audience Q&A and Discussion (Moderated by Abby Bronson, SVP Research Strategies, PPMD)

3:15 – 3:30 Closing Thoughts and Next Steps

Tim Franson, MD, YourEncore


Advocates head to the hill

No breakfast served on Tuesday am

Advocates head to Capitol Hill for House and Senate Meetings.

Transportation instructions via metro or cab can be found in advocate packets.

All Advocates will be given schedules for meetings via email and on Sunday.

12 Noon – 1pm: PPMD Congressional Hill Briefing

Realizing the Promise of Patient-Focused Drug Development. (Box lunch will be served.)

Where: Room – Senate Visitors Center 202-203. (The Capitol Visitor Center, the main entrance to the U.S. Capitol, is located beneath the East Front plaza of the U.S. Capitol at First Street and East Capitol Street, across from the Supreme Court. Please be in the line for Security by 11:15 AM)

All advocates invited to attend as long as it doesn’t conflict with a Hill meeting.

By Tim Gillen

I'm a born-again christian, sold out to serving the needs of the less fortunate and in Sub-Saharan Africa. I founded The Everett and Austin Project in October 2018 to honor my 2 boys posthumously by helping people living in poverty going thru Duchenne Muscular Dystrophy and other rare diseases. My sons each died of Duchenne, Austin in 2012 at age 16 and Everett in 2017 at age 22.

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