Our vision is our name:
CureDuchenne was founded by Paul and Debra Miller in 2003 after their son was diagnosed with Duchenne muscular dystrophy. The Millers applied their professional backgrounds to create a fiscally responsible and strategically focused business model, and assembled a seasoned staff and an expert Scientific Advisory Board, to lead the charge for a cure. Today, our nonprofit is a national leader in affecting change worldwide.Read message from the founders
What is Duchenne?
Duchenne is a devastating muscle disease in children. Historically, most boys who have it do not survive beyond their mid-twenties, and those that do will be wheelchair bound by age 12 and experience social isolation. The simplest of tasks become difficult, and in the later stages, heart and breathing muscles begin to fail. Nearly 20,000 boys are living with the disease in the United States alone and over 300,000 worldwide. The disorder knows no cultural, economic, or social boundaries.
Muscular dystrophies are a group of genetic disorders characterized by muscle weakness. Duchenne is the most common and severe form, caused by loss of dystrophin, beneath the sarcolemna. The molecular mechanisms of the disease have been extensively investigated since the discovery of the gene in 1986.
The good news is that new gene-based therapies have recently emerged with noted advances in using conventional gene replacement strategies, RNA-based technology, and pharmacological approaches. In particular, antisense-mediated exon skipping has shown encouraging results and holds promise for the treatment of dystrophic muscle.
Mission and Vision
Our efforts are targeted. We have the leading scientists in the world helping us determine the most viable research projects, accelerate the clinical trial process and bring potential life-saving drugs to help this generation of Duchenne boys.
Our vision is our name…to cure Duchenne muscular dystrophy.
Our mission is to save this generation of Duchenne boys.
With increased awareness and increased funding we’ll extend and improve the lives of those afflicted with Duchenne.
The Words of a Duchenne Parent
I was so excited when I found out I was expecting a baby and going to be a mother for the first time. When I saw him for the first time, I just fell in love with my perfect little baby boy, Ian Michael Schneider. He was such a great baby – he slept, ate, laughed and had the chubbiest cheeks and the brightest eyes.
Three years later, our lives changed forever in a matter of an hour when my husband, Michael, and I were thrown into the world of Duchenne Muscular Dystrophy. We learned Ian will not live a full life and our family will have to watch him suffer as the disease progresses.
I often think, “I did this to Ian.” Not intentionally, I know, but I still passed it to him.
Ian just turned 9 and things are very different for him and the rest of our family. Instead of being fitted for a football uniform, he is being fitted for a motorized scooter. I hate to watch Ian frustrated when he falls down, when he struggles in school with physical and academic issues, when he can’t do anything but be carried on daddy’s shoulders at his cub scout camping weekend, when he has no one but us to play with, when he has to sit in a wheelchair on his class trip because there is walking involved.
My heart breaks when I have to give him a growth stunting steroid every day, when we feel out of place among healthy families or shut out completely because they can’t relate, when we dread the day the words “I can’t walk anymore” will come out of Ian’s lips, when the only thing on my mind is how long will we have our son and when will Duchenne take everything from him and our family?
By Donna Schneider, Phillipsburg, New Jersey
Message from the Founders
Imagine having a beautiful baby boy. For four years, your child thrives and is the light of your life. One day, you notice he has difficulty standing up. His calves are swollen, and he can’t keep up with other children. You take him to his pediatrician, who says he’s probably just a slow starter and not to worry.
At five, you finally find a specialist that gives a proper diagnosis: Duchenne muscular dystrophy. You’re told his muscles will die and not regenerate. You scream frustration as to why you haven’t heard about Duchenne before. Then, you find out pharmaceutical companies do not invest in it because the number of potential users does not make it profitable enough.
That’s our experience, and for the love of our child, we’ve taken matters into our own hands and embarked on a path to affect change: to raise the funds needed to find a cure within our son’s lifetime. We’ve always had faith, but we never knew how much we would come to rely on it. We hope all Duchenne families are provided the comfort needed to get through these difficult times.
We are thankful to our partners and sponsors for sharing our vision and collaborating with us to find a cure. Saving the children affected by Duchenne has become our life’s work, and we hope you’ll join with us.
Paul and Debra Miller
Debra Miller, President & CEO
Debra Miller co-founded CureDuchenne in 2003 with her husband, Paul, after their only son was diagnosed with Duchenne. Miller relies on her extensive background in sales and marketing to lead CureDuchenne.
Her role includes overseeing all operations, research and financial resources as well as serving as the primary liaison to the Board of Directors. She is the decision maker, leader and manager in carrying out the mission of the organization. She also works closely with the Scientific Board of Advisors in designing and implementing the medical research strategic plan.
Miller earned a Bachelor of Arts in Communication Studies from the University of California Los Angeles. Prior to CureDuchenne, she had a career in publishing with positions in advertising sales and was an independent stock trader.
Jak Knowles, MD, Managing Director, CureDuchenne Ventures and Vice President Medical and Scientific Affairs CureDuchenne
Dr. Jak Knowles joined CureDuchenne in 2015 as the Managing Director of CureDuchenne Ventures and the Vice President of Medical and Scientific Affairs for CureDuchenne. Dr. Knowles has had a career in healthcare spanning over a decade with the last five years focused in biotech venture capital and finance. Most recently, Dr. Knowles was an investment professional in the Boston office of MPM Capital ($2B AUM) where he worked with the BioVentures Asset Management team to create early-stage companies focused on developing curative therapeutics across a wide spectrum of indications.
Prior to MPM, Dr. Knowles worked in New York as a biotechnology equities analyst within the investment bank JMP Securities, and with Burrill & Co., a life sciences VC firm. Dr. Knowles began his post-graduate business career working in health and life sciences consulting practice of Oliver Wyman where he helped implement value-based transformation of hospital systems and physician practices through re-designing care-models, implementation of electronic clinical decision support, and structuring aligned re-imbursement based on patient centric care.
Dr. Knowles has a medical degree from Stanford University where he graduated with honors in biomedical research, and a B.S. in biology from Binghamton University. Dr. Knowles has authored over a dozen research publications from work performed at the Memorial Sloan Kettering Cancer Center, Stanford University, and as part of a NIH sponsored post-doctoral surgical research fellowship.
Michael Kelly, Chief Scientific Advisor
Dr. Michael Kelly joined CureDuchenne in December 2011 as chief scientific officer. Kelly, a senior pharmaceutical executive, brings more than 25 years experience in drug discovery and development to the organization. He is responsible for advancing drug development programs and identifying new drug targets that exhibit potential to transform the treatment of Duchenne muscular dystrophy.
Before joining CureDuchenne, Kelly served as president and U.S. site head of Renovis, Inc. and has held senior positions at Amgen, Inc., Wyeth (Pfizer) and Wellcome (GlaxoSmithKline).
Kelly holds a Ph.D. from the University of Southampton (U.K.), held research positions at the University of Michigan and University of Nottingham, is an inventor on more than 100 patents and is widely published in peer reviewed journals.
Karen Harley, Vice President of Marketing Communication
Karen Harley joined CureDuchenne in December 2011 as director of marketing communications. She has more than 20 years experience in public relations, social marketing and public education. She is responsible for marketing, communications, public relations, special events, website and building partnerships to help increase awareness about Duchenne muscular dystrophy and raise funds to find a cure for Duchenne.
Before joining CureDuchenne, Harley was director at PainePR. Previously she was vice president of Hill & Knowlton. Harley has managed large-scale public education campaigns focused on obesity prevention, service and volunteering, teen pregnancy prevention, mentoring and disaster preparedness that helped drive behavior change. She has worked with a variety of clients in the public, private and nonprofit sector.
Harley holds a bachelor of arts degree in English and psychology from Indiana University in Bloomington, Indiana.
Drew Hoyer, Vice President of Development
Drew Hoyer joined CureDuchenne in April 2013 as Senior Director of Development. He is responsible for overseeing all fundraising and cause-related marketing efforts. His role includes donor stewardship, cause-related marketing initiatives, sponsorships and board development. He brings more than 7 years of experience in development, philanthropy and community relations to the organization.
Hoyer was the Senior Director of Development for the Cystic Fibrosis Foundation Orange County Chapter. Hoyer has managed large scale fundraising events for the CFF and worked with many families to help find a cure for CF. He now brings this experience to CureDuchenne to help us reach our goal of a cure.
Hoyer holds a bachelor's degree in Science and Youth Ministry from Martin Luther College.
Rachel Nelson, Director of Development
Rachel Nelson joined CureDuchenne in November 2014 as director of development. She is responsible for parent outreach, special events, volunteer management, and donor cultivation. Over the last ten years her career has focused on business development and marketing.
Nelson comes to us from Mark Beamish Waterproofing where she was a project estimator specializing in sales for restoration projects. Previously, Nelson served as director of business development for the Muscular Dystrophy Association Greater Orange County Chapter. During her tenure at the MDA, she identified and built partnerships with new sponsors as well as leading strategic fundraising initiatives, media campaigns and volunteer outreach.
Alicia Clough, Database Manager
Alicia Clough joined CureDuchenne in May 2013. Clough is responsible for office operations, data entry, donor database/ CRM management and event coordination. She gained two years of office experience in Jacksonville, FL.
Clough holds a Bachelor of Science degree in sociology from Jacksonville University.
Katie Mastro, Program Services Manager
Katie Mastro joined CureDuchenne in September 2015 as program services manager. She is responsible for managing and expanding the CureDuchenne Cares program, engaging with families whose loved ones have Duchenne Muscular Dystrophy, and coordinating events.
Mastro comes to us from Global Genes, a patient advocacy organization that offers support and education to the broader rare disease community. While there, Mastro helped build an amazing library of educational toolkits and webinars that are valuable resources to the patient community. In addition, she managed the Corporate Alliance and its committees, creating an engaged and committed community of corporate supporters providing value for both them and the organization.
Mastro holds a bachelor of arts degree in history with a minor in political science from the University of California, Los Angeles.