In January of 2014 we held our inaugural PAAC Leadership Workshop in Washington,DC. The day included engagement with federal policy makers, congressional staff, and research leaders. At the end of the day, a long-time colleague of mine from the FDA who had spent the entire day with us pulled me aside and said, “Wow. These men are brilliant. They have such important insight about clinical trials and public policy. But Annie… before today, I never knew that there were ADULTS with Duchenne.”
Yes. This was exactly IT! This was the entire purpose of the PAAC. To have ADULTS with Duchenne/Becker raise the priorities of the emerging (and growing) adult Duchenne/Becker community.
In just 3 years, the PAAC has helped to formalize the voice of the emerging adult Duchenne community. We have engaged regulators, policy makers, industry, and Congress – and members of the PAAC now serve as patient representatives to industry DSMBs, NIH Councils, FDA’s patient representative program, the CDC’s Care Considerations workgroups, and more. Our PAAC has informed PPMD’s efforts with outside partners such as our work with the ABLE implementation, and have helped to led initiatives such as PPMD’s robotics initiative and our mental health study. We have influenced representation on FDA Advisory Committees and labeling.
Simply put, the PAAC is changing the landscape. And we are thrilled to share that 2018 is shaping up to be an even more productive year for the PAAC than ever before.
Before I introduce the 2018 PAAC, I’d like to take a moment to recognize the members of the 2017 PAAC who will not be returning to us in 2018.
A Debt of Gratitude To Those Who’ve Served Our Community
While Mario will not serving in a formal leadership capacity for PPMD in 2018, he will no doubt continuing to lend his experience and mentorship to our growing community. Mario has been a close personal friend of mine for nearly 2 decades which makes it even more wonderful to share that the reason he won’t be returning to the PAAC this year is because he and his wife, Theresa became parents to their beautiful daughter Petra this past fall – and Mario will be devoting more time to his family in 2018.
PPMD and our Duchenne/Becker community are grateful to Mario for his leadership of our PAAC. Mario and his wife Theresa have been strong contributors to the PPMD community. For the past 3 years, he has generously lent his professional expertise as an attorney specializing in compliance with federal regulations related to physical and disability accommodations. Through his role on the PAAC, Mario has helped to shape critical policy initiatives and has served on the planning committees for PPMD’s Transitions Meetings. In 2016, Mario served as the Chairperson of the PPMD Adult Advisory Committee.
Very tragically, our PAAC lost two of our leaders in 2017.
In 2017, PAAC members Susie Arroyo and Mohammed Haider transitioned from community leaders to powerful legacies.
While losses of young adults with Duchenne are difficult for our entire community, their deaths deal a particularly devastating blow to our PAAC members who grow to share a very personal bond with one another.
Within our PAAC, we choose to honor these men and women by helping to build their legacies; prioritizing projects and policies that held great meaning to them. Our Duchenne community will continue to see the impact and love of these amazing leaders shine through in the efforts of the PAAC throughout 2018 and beyond – just as we have worked to ensure that Ben Cumbo and Matt Petrusko’s legacy’s continue to live on.
Susie was a twenty-eight year old woman with Duchenne Muscular Dystrophy from Pendelton, Oregon. She dedicated her life to her community, volunteering with nursing homes, serving as a mentor to young children and teens, and working within her church community. In addition to serving on PPMD’s Adult Advisory Committee, Susie also served on the board of the Clearview Mediation and Disability Resource Center. Susie held a Bachelor’s degree in Elementary Education and Multidisciplinary Studies from Eastern Oregon University. Susie was passionate about awareness that womancan be manifesting carriers of Duchenne and have the Duchenne phenotype – and wanted to see that reflected in our community’s terminology and priorities. PPMD worked closely with her on these issues, incorporating language around the need for broad labeling into the Duchenne community-led Guidance for Industry that ultimately led to a broad label once the first Duchenne therapy was approved. This was a banner moment for Susie and all girls/women with Duchenne globally. Susie was amenable to Exondys51 and was able to access the commercially approved therapy in her lifetime. PPMD & the PAAC will continue to ensure that her legacy lives on.
Mo was a twenty-eight year old who served as an unwavering advocate for our Duchenne community on all levels for decades. Through his participation in PPMD-led efforts such as the Draft Guidance for Industry and the development of the patient preference survey instrument, Mo inserted the critical perspective of the adult community. Mo also worked to advance therapy development through both his academic pursuits and internship experiences including work under Dr. Hansell Stedman, as well as personal participation in interventional studies. Mo lived independently in Philadelphia, held a BA in Economics & International Relations from the University of Pennsylvania and a masters degree in Finance from Vilanova School of Business. Mo was passionate about ensuring that adults with Duchenne had access to independent living options and were aware of technologies to enable autonomy. In his honor, the PAAC will be completing and publishing a resource guide in early 2018 that Mo had been eager to see developed and disseminated.
Welcoming PPMD’s 2018 Adult Advisory Committee
As we look at the year ahead, the Duchenne landscape is filled with unparalleled opportunities. 2018’s landscape also includes an unpredictable access and policy environment making it even more crucial that we have an adult advisory committee comprised of Duchenne/Becker experts who are well positioned to provide leadership and guidance to PPMD as we navigate the challenges and opportunities before us. I am thrilled to introduce the men and women who comprise the 2018 PPMD Adult Advisory Committee. The 2018 PAAC includes members with diverse academic and professional backgrounds including law, accounting, engineering, finance, sports broadcasting, education, literature, biochemistry, communications, international affairs, management, and more. They are men and women from around the nation who whose common thread is a shared commitment to contributing to PPMD’s Duchenne/Becker community. And together, they are changing our world.
Please join us in welcoming our 2018 PAAC – and thanking them for leaning in to help shape the Duchenne landscape.
* Joining PPMD Adult Advisory Committee (PAAC) in 2018
Dx: Manifesting Carrier, Duchenne
Home state: New Jersey
After the sudden death of her brother and former PAAC member Matt Petrusko. Jenn joined the PAAC to help continue his extraordinary legacy. A devoted mom and wife with a background in business, Jenn’s passion and expertise have been a strong complement to the team. In addition, as a manifesting carrier with significant muscle weakness, she has been able to help represent the priorities of female members of our Duchenne/Becker community.
“I first joined the PAAC last year as an outlet for my grief. At first, I was nervous about how a manifesting carrier would fit in. The members of the PAAC were so welcoming and I saw a part of Matt in each and every one of them. I was absolutely blown away by all the wonderful, smart, caring and talented men that are a part of the PAAC. I also was so impressed to meet a lot of their family members. We all are part of a larger group, people that are fighting the beast of BMD/DMD. I feel like I belong, I know Matt is guiding me though this and I am so thankful that I was given this opportunity.” ~ Jenn Bauer
Hometown: Annandale, Virginia – currently living in Irvine, California
Buddy has been involved in advocating for and bringing public awareness to the challenges and successes of those with physical disabilities, particularly neuromuscular diseases, since he was a little boy. His advocacy efforts formalized as a student attending the University of Richmond when he would regularly meet with administrators on how to improve the accessibility of the campus for those with impaired mobility. His educational pursuits led him to a study abroad program at Oxford University and then back to the U.S. where he received an MFA from American University. Buddy previously worked as a docent at the Folger Shakespeare Library in Washington, D.C. and currently is attending the University of California Irvine where he is completing a PhD program in English literature.
As a member of the PPMD Adult Advisory Council, Buddy has testified at congressional briefings, helped to shape policy, and has served as a mentor to others within our community. In 2015, his work with the PAAC brought him in contact with the FDA where he learned of the FDA’s Patient Representative Program. Buddy now serves as a special government employee to the FDA, representing the Duchenne community.
Home state: Massachusetts
Alan Chaulet was born an entrepreneur and political advocate. With a degree in Management from Bentley University, Alan immediately launched his first business called ‘Badass Ability’. Over the last 2 years, Alan has been making a federal policy impact as co-founder of All Wheels Up, an organization working with federal aviation officials and engineering professionals to enable passengers remain in their wheelchairs during air travel. Alan also works as the adult community advisor to PPMD’s DuchenneConnect. He is incredibly passionate about improving the drug development process and ensuring healthcare access for all with Duchenne and Becker.
Hometown: Canton, MA
Michael is currently a student at the University of Massachusetts Boston, studying communications and sports broadcasting. Diagnosed with Duchenne at age 3, Michael has been dedicated to serving others. His community engagements have included work as a peer leader at Partners Youth with Disabilities, and working as a counselor for the Easter Seals Camp for children with Autism. In addition to attending college, Michael works with Solid Biosciences in Cambridge, Massachusetts.
Hometown: Fort Mill, SC
Sean is an avid sports enthusiast and is often found announcing local sporting events and announcing for the Victory Sports Conquer League – but the sport he is most passionate about is football (and he’s a diehard Panthers fan). Sean also enjoys playing chess and Xbox, listening to music, watching movies, meeting new people, and memorizing various stats and facts.
Sean is also very committed to contributing to his community and spends three days a week volunteering with adults with cognitive disabilities leading activities, small groups, and serving as a mentor at the Adult Enrichment Center. He also serves as an assistant chess coach for a local elementary school.
Sean is employed with ABLE South Carolina and serves on their adult advisory council.
Hometown: Dallas, Texas
Ben is a valued member of the PPMD community whose passion for Duchenne advocacy started at an early age as he met with members of Congress while in middle and high school and advocated for the passage of the MD-CARE Act. In 2015, 2016, & 2017, Ben served as a panelist and organizer of PPMD’s Transition meeting in conjunction with the Annual PPMD Connect Conference and was a passionate advocate during the EndDuchenne Hill Rally that June. Ben joined the PAAC in 2016 and has been making a significant impact.
Ben’s work with the PAAC brought him in contact with the FDA where he learned of the FDA’s Patient Representative Program. Ben now serves as a special government employee to the FDA, representing the Duchenne community. Ben also works with PPMD’s PCORnet program and is helping to lead a Mental Health Initiative within our Duchenne Community, in connection with DuchenneConnect and the PAAC.
Ben has a degree in Biochemistry from Southern Methodist University and is pursuing a graduate degree in Genetic Counseling. He has also been involved in early exploration of the CRISPR Cas-9 study and it’s application to Duchenne.
Home state: Maryland
Brett was born and raised in northern New Jersey and was diagnosed with Becker muscular dystrophy at age 9. Brett received his B.S. in Justice Studies from Arizona State University in 2003 and his J.D. from the University of Texas School of Law in 2007. After graduating law school, Brett clerked for the Honorable Diane M. Johnsen on the Arizona Court of Appeals before moving to Baltimore to work as the inaugural Disability Rights Fellow at Brown, Goldstein & Levy, LLP. He currently works as an Assistant Attorney General in the Maryland Office of the Attorney General, where he provides a wide variety of advice and litigation services for the Boards that regulate the health occupations and license health professionals in Maryland. Brett previously served on the Board of Directors of the Maryland Disabilities Forum, and he currently sits on the Board of Directors of the American Civil Liberties Union of Maryland and serves as the Board’s Affirmative Action Officer. He lives in Baltimore City.
Hometown: Arlington, Massachsutts
Austin has been an active member of the Duchenne and PPMD community for much of his life – but has become a dedicated advocate within the last 5 years. Austin is passionate about clinical trial access and has had extensive personal experience with clinical trial participation and overcoming barriers to therapies. He enjoys public speaking and has testified both before the FDA and within Congressional Briefings on behalf of our Duchenne community.
Austin is eager to join the PAAC to be able to serve as a mentor to others with Duchenne and their families – and to help to shape the policy priorities of our community. Austin possesses great skills in computer technology and 3D printing and has conducted numerous internships and projects in this technical skill area; he is currently a freshman at Massasolt College pursuing a degree in Mechanical Engineering.
Hometown: Plantation, Florida
Alex is a journalism major in his junior year at Broward College in Davie, Florida and is pursuing a life-long dream to become a sports journalist. Having been diagnosed with Duchenne at age 2 ½, he has been an integral part of the Duchenne community and attended PPMD’s Transitions Conferences and PAAC-led events in recent years, he is eager to serve as a leader within the PAAC and help guide policy for teens and adults within our community. Alex is a fantastic sports blogger.
Hometown: Penfield, NY
Jonathan refuses to allow Duchenne to define who he is and has sought every opportunity to push boundaries and exceed expectations placed upon him. His achievements include acquiring the rank of Eagle Scout, graduating from Nazareth University with a degree in Biology and minors in Chemistry and Toxicology, and volunteering at both the Rochester Museum of Science Center and the Seneca Park Zoo. Jonathan drives a van, modified with joy stick controls. He also has actively participated in clinical trials, natural history studies, and investigational studies all aimed at supporting therapy development for Duchenne, and has met with Congressional representatives and regulators to advance PPMD ‘s Duchenne community initiatives. Jonathan is a tremendous advocate and contributor to the Duchenne community.
Hometown: Richmond Hill. Ontario
Kanch has spent his professional life working in both the public and private sectors, while maintaining a focus on raising disability awareness within each sector and in the community at large. His professional and volunteer roles have included the Bank of Montreal Diversity Council, Access-York University, Able-York, and the Centre for Independent Living in Toronto. Kanch has a political science degree from York University in Toronto.
Kanch has been married for 14 years and has 3 children. In 2013, he had a tracheotomy performed, and uses a ventilator at night.
Kanch and his family has been impactful contributors to PPMD’s community through his involvement on the PAAC. Attending conferences, leading Transitions meetings, and shaping policy initiatives, he has infused his knowledge and experience of living with Becker’s into the legislative and policy initiatives of PPMD.
Hometown: Kalamazoo, MI
Colin has an extensive background in advocacy and service, having contributed extensively to his college, church, and local communities for many years. An Andrew W. Mellon scholar at Hope College, Colin graduated with degrees in both music and history and numerous noteworthy accomplishments as a musician throughout his college career. As a member of PPMD’s Adult Advisory Committee, Colin has helped to shape federal policy, co-led PPMD’s national transitions conference, and participated in federal advocacy efforts on Capitol Hill. When asked about his experiences with the PAAC over the last year he reflected, “we all come to the table having experienced life with Duchenne or Becker, and it is awesome to be able to work together to tackle this disease”.
Colin spent several months in 2017 pursuing his passion and expertise for music in Vienna, Austria.
Hometown: Lincoln, Nebraska
Alex is a senior at the University of Nebraska Lincoln studying biological sciences. Following graduation, he plans to become involved in biomedical research to find treatments for diseases such as Duchenne muscular dystrophy.
Alex is particularly passionate about utilizing assistive technology to overcome physical barriers. He has personally used assistive technology in such a way that has allowed him to be successful despite requiring a wheelchair full time for mobility and having limited use of his hands. With this experience, he would like to help other adults living with Duchenne muscular dystrophy.
Hometown: Meriden, CT
Josh and his family have been active members of our PPMD community since he was first diagnosed with Duchenne at the age of one. Like many within our community, his diagnosis has waivered between Duchenne and Becker as diagnostic criteria and clinical symptoms progressed, but what never changed was his commitment to the community. Josh has participated in the Ataluren clinical trial since the age of 12 and in the fall of 2017 testified on behalf of the product’s approval during the Open Public Hearing at the FDA Advisory Committee.
Josh graduated with an accounting degree from Pace University in New York, NY and is currently pursuing his graduate degree in accounting at Southern Connecticut State University. In his application for the PAAC, Josh stated “I want those affected by either DMD or BMD to be able to live their lives with more medical options and opportunities… I feel that I can offer a unique perspective on Becker Muscular Dystrophy, and help advocate on behalf of Duchenne and Becker Muscular Dystrophy. “
Hometown: Farmville, Virginia
Colin graduated from community college in May of this year and works as an administrative assistant for Letterpress Communications. He also dedicates time volunteering as an assistant superintendent for a local model railroad club.
Colin joined the PAAC in 2016 after having been involved with PPMD since he was first diagnosed with Duchenne in 1998. While his family has been involved in all levels of the community and organization, being a part of the PAAC allowed Colin to advocate as an adult within our community. He has spoken at Every Single One tour venues, participated in PAAC-led meetings, advocate on Capitol Hill, and now is working with an industry partner to integrate the perspective of the adult community in their drug develop and access program considerations.