Muscular Dystrophy Association Presents MDA Tribute Awards in Washington D.C.
Muscular Dystrophy Association Presents MDA Tribute Awards in Washington D.C. to Honor Diana X. Bharucha-Goebel, M.D., Associate Professor, Pediatrics and Neurology, Children's National Hospital, R. Rodney Howell, M.D., Worldwide Leader of Newborn Screening, Paul Robertson, Founder of Fishing For Muscular Dystrophy, Jeni Stepanek, Ph.D., aka Mama Peace, Advocate, Author, Educator Janet Woodcock, M.D., Principal Deputy Commissioner, Food and Drug Administration
MDA Tribute Tour in Washington, D.C. includes raising awareness with Major League Baseball for Lou Gehrig Day with the Washington Nationals vs. Phillies June 2.
NEW YORK, NY – May 10, 2023 – Muscular Dystrophy Association (MDA) Tribute Awards will be held in Washington D.C. to honor: Diana X. Bharucha-Goebel, M.D., Associate Professor, Pediatrics and Neurology, Children's National Hospital; R. Rodney Howell, M.D., worldwide leader of newborn screening, Professor and Chairman of Pediatrics, Emeritus, Hussman Institute for Human Genomics, University of Miami, Miller School of Medicine, Emeritus Member and former Chairman of the Board of Directors of MDA; Paul Robertson, Founder of Fishing For Muscular Dystrophy; Jeni Stepanek, Ph.D., aka Mama Peace, MDA family member and award-winning speaker and writer, as well as a noted advocate for personal and world peace and children's and families' needs and rights in health and education; and Janet Woodcock, M.D., Principal Deputy Commissioner, Food and Drug Administration, an advocate for American patients and consumers, working to help the Commissioner develop and implement key public health initiatives. The MDA Tribute Award recipients have all contributed to significant progress in accelerating research, advancing care, and advocating for the support of people living with neuromuscular diseases. The MDA Tribute Awards presentation and reception will be held on Saturday, June 3, 2023, from 6-8:30pm at The National Press Club.
The MDA Tribute Awards in the nation's capital are notable because they honor area individuals who have made extremely significant contributions to progress for people living with neuromuscular diseases through scientific discovery, care, and fundraising. They are presented to acknowledge the legacy and life's work of those whose extraordinary efforts support the patients and families MDA serves.
The MDA legacy represents the contributions of so many pioneers who, through their work, have changed the trajectory for neuromuscular disease. This event celebrates leaders in the Washington, D.C. community who led progress and collaboration, with their commitment to bringing cures and treatments to all people living with muscular dystrophy and related neuromuscular diseases.
Throughout MDA's 73-year history, local volunteers and communities have continued to be at the heart of the organization's mission to empower the people it serves to live longer, more independent lives. The event celebrates strength in community and strength in unity of Washington, D.C.'s volunteers and partners that help make the MDA the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases.
"We are turning the spotlight on Washington, D.C., to pay tribute to capital-area physicians, researchers, advocates, and allies who have dedicated their careers to bring this extraordinary moment in time to bear for people living with neuromuscular disease. Thanks to their distinguished contributions, hope for longer more independent life is a reality for people with neuromuscular disease. The Muscular Dystrophy Association Tribute Tour in Washington, D.C., continues the tradition of honoring people at the heart of communities across the U.S. who are generating unprecedented breakthroughs in science, regulatory policy, access, inclusion, and quality of life for families living with muscular dystrophy, ALS, and related neuromuscular diseases. Our Washington, D.C. honorees stand apart in their respective commitments to finding treatments, making them accessible, and amplifying the voices of our MDA community members," said Donald S. Wood, Ph.D., President and CEO, MDA.
About MDA Tribute Tours and Awards
MDA Tribute Tours have celebrated Strength in Unity, Strength in Community in Las Vegas, Nashville, Houston, St. Louis, Boston, and now Washington, D.C. This platform provides an opportunity for the community to come together in a show of strength, gratitude and admiration for these extraordinary individuals who have been tireless in their efforts to support the families MDA serves. The honorees selected are the embodiment of the progress in neuromuscular disease research and care for over 73 years – from families to clinicians and researchers working together with the scientific community at large, with the shared mission to empower people living with neuromuscular disease to live longer more independent lives.
MDA Tribute Awards: Washington, D.C. Recipients
Diana X. Bharucha-Goebel, M.D. is the MDA Care Center Director at Children's National Hospital in Washington D.C., Professor in the Division of Pediatrics and Neurology at the George Washington School of Medicine and Health Science and a clinical researcher in collaboration with the National Institutes of Health (NIH) in the Neuromuscular and Neurogenetic Disorders of Childhood Section. Her interest in neuromuscular medicine began in 2000 when she first volunteered with the MDA Summer Camp program. She completed her medical training at Drexel University College of Medicine, followed by Pediatric Residency, Child Neurology Residency and Neuromuscular Medicine Fellowship at Hospital of Philadelphia and Hospital of the University of Pennsylvania. Following her clinical fellowship training, she began research at the National Institutes of Health (NIH) in Bethesda, Maryland and concurrently joined the Division of Neurology at Children's National. Her research has focused on the development of clinical outcome measures and biomarkers for a rare neurodegenerative disorder, giant axonal neuropathy (GAN). She has worked with Dr. Carsten Bonnemann at NIH on the development of a first-in-human intrathecal gene transfer trial for GAN. Her primary clinical and research interests lie in the development of early phase gene therapy focused trials for patients with rare neuromuscular disorders. In addition, her research interests have also focused on studying the immunologic consequences of viral mediated gene transfer, and the role of immune modulation in dampening these responses and improving safety of gene replacement approaches. Clinically, she leads an expert multidisciplinary team at the Muscular Dystrophy Association Care Center at Children's National Hospital, and is involved in the diagnosis, care, treatment, and research for children with a wide range of neuromuscular conditions.
"It's an honor to be recognized by the Muscular Dystrophy Association for my work at MDA Care Center Children's National with our multidisciplinary team which is so critical for the care of people living with neuromuscular disease," said Dr. Bharucha-Goebel. "Our team has long strived to provide comprehensive and specialized care for children with muscular dystrophy and related neuromuscular diseases, ranging from expertise in genetic and electrophysiologic diagnostics to specialized family centered care in orthopedics, physical and occupational therapies, physical medicine and rehabilitation, neurology, nutrition, and bone health. We are excited for the ongoing opportunities as an MDA Care Center, especially at a time when novel therapeutic strategies are emerging in the field of neuromuscular medicine, such as for spinal muscular atrophy, Charcot-Marie-Tooth, Pompe, and Duchenne muscular dystrophy."
R. Rodney Howell, M.D., FAAP, FACMG is the founding chair of the congressionally-mandated Secretary's Advisory Committee on Heritable Disorders of Newborns and Children and an advisor to the director of the National Institute of Child Health and Human Development (NICHD). Dr. Howell joined Muscular Dystrophy Association's (MDA) Medical Advisory Board in 1981. After serving as Chair of MDA's Scientific Advisory Board, he joined the MDA National Board of Directors and later served as Chairman. In 2022, MDA elected Dr. Howell as its first ever Emeritus Chairman of the Board. He is Professor and Chairman of Pediatrics Emeritus at the Miller School of Medicine at the University of Miami, and an elected fellow of the American Association for the Advancement of Science. The author of more than 150 original articles and abstracts on genetic disorders in children, Dr. Howell's pioneering work has benefited countless infants and children worldwide. Dr. Howell's skill in advancing the genetic and genomic medicine agenda has been pivotal in improving global public health.
"I'm honored to be recognized for my work by an organization that has been the foundation for neuromuscular disease innovation and has always inspired my focus on newborn screening as one of the most important and impactful public health programs in the United States," said Dr. Howell, former MDA Chairman of the Board of Directors and Professor of Pediatrics and Chair Emeritus of the Department of Pediatrics at the Miller School of Medicine at the University of Miami. "Since its inception, newborn screening has saved and improved the lives of thousands of children. Continued expansion will allow for screening for more neuromuscular conditions such as Duchenne muscular dystrophy (DMD). This will benefit many more families by enabling them to receive the care their children need from day one. This is especially important because in many cases of progressive degenerative diseases, what is lost cannot be regained. Newborn screening is a catalyst for the study of rare diseases and is a major driver for progress in rare disease and genetic research."
Paul Robertson is the is the Founder of Fishing For Muscular Dystrophy, a native of Maryland and successful entrepreneur and businessman, who has been diagnosed with Muscular Dystrophy (MD). Paul was diagnosed with Limb-Girdle Muscular Dystrophy (LGMD) nearly 20 years ago, but that has not stopped him. After dealing with mobility challenges, Paul decided it was time to not only become a champion and spokesperson for muscular dystrophy, but also use his talent and passion for fishing and align it with his dedication to increasing research funding, awareness, and support for the Muscular Dystrophy Association (MDA). In 2015, Paul founded the Fishing for Muscular Dystrophy Foundation (FFMD) to partner with the MDA and has raised over $3 million for the mission.
"It's an honor to be recognized with the MDA Tribute Award for the important work we're doing strategically with Muscular Dystrophy Association that is leading to incredible progress in gene therapy and support for people living with LGMD and neuromuscular community at large," said Mr. Robertson. "When we started FFMD, we wanted to engage other corporate entities as sponsors and affiliates including but not limited to put their logos on the side of our boat and further broaden the awareness and support of MDA and those affected with muscular dystrophy. We've adapted that model over time, and it's grown into so much more." Today the organization has expanded beyond competing in fishing tournaments. FFMD also raffles off boats, helps to put on Muscle Team galas, sells merchandise and is involved in several other initiatives that raise money and awareness for neuromuscular disease. "Ultimately, we want to be responsible for a $1 million-per-year contribution to MDA and it's only a matter of time."
Jeni Stepanek, Ph.D. also known as "Mama Peace" is a Senior Faculty Specialist, University of Maryland, President and Chief Peace Ambassador of the Mattie Stepanek Peace Foundation. She is a bestselling author, well-respected educator, and advocate. Dr. Stepanek is an award-winning inspirational speaker, but she is most proud of her purpose as a memory curator, peace cultivator, and "welcome mat." As a child in the 1960s, she watched the Telethon each Labor Day, and thought, "when I grow older, I want to help these children and families and medical teams. I want to be a part of the support, of the hope." She participated in backyard carnivals and community fishbowl fundraising events and pledged her annual support to make a difference, never imagining she would be on the other side of the support, or the hope. Dr. Stepanek lives with neuromuscular disease and parented four children whom she unknowingly passed a mutation of her condition before being diagnosed herself. Her youngest, Mattie Stepanek, was an MDA National Ambassador who met with world leaders and celebrities alike to raise awareness of hope and peace. He regularly attended MDA Summer Camp for a week of "heaven on earth" and like his mother, has always supported MDA's partnership with the International Association of Fire Fighters to "Fill the Boot." Dr. Stepanek's son Mattie was a high-profile MDA National Goodwill Ambassador during his final three years, making an incredible impact to raise awareness.
"MDA has long been a part of the fabric of my life journey. I could never have imagined that one day, when I grew older, that I would be honored by MDA – for a life journey that moved me from one side of the story of hope as a viewer, to the middle of the story as a patient and parent, and to the ultimate essence of the story of hope as an advocate. We can't always plan the details or the facts of our life journey. But we can choose attitudes and actions that reflect the powerful truth of hope in any journey. We can choose to let the facts shape us, but not define us. We can choose to live such that the tough stuff strengthens us rather than hardens us. We can choose to be a gift of hope – the thread that connects and supports and strengthens – on any side of any story. I am grateful, and I am proud!" said Dr. Stepanek.
Janet Woodcock, M.D., is the Principal Deputy Commissioner - Office of the Commissioner, Food and Drug Administration. In this role, she works closely with the Commissioner of Food and Drugs to develop and implement key public health initiatives and helps oversee the agency's day-to-day functions. She served as the Acting Commissioner of Food and Drugs from Jan. 20, 2021, until Feb. 17, 2022. Dr. Woodcock began her FDA career in 1986 at the Center for Biologics Evaluation and Research (CBER) where she served as the Director of the Division of Biological Investigational New Drugs and as Acting Deputy Director and Director of CBER's Office of Therapeutics Research and Review, which oversaw the approval of the first biotechnology-based treatments for multiple sclerosis and cystic fibrosis during her tenure. In 1994, she was named Director of the FDA's Center for Drug Evaluation and Research (CDER), leading the Center's work that is the world's gold standard for drug approval and safety. In 2004, she became the FDA's Deputy Commissioner and Chief Medical Officer. Later she took on other executive leadership positions in the Commissioner's Office, including Deputy Commissioner for Operations and Chief Operating Officer.
"It's an honor to receive the Muscular Dystrophy Association Tribute Award, from an organization that has been the leader in developing the field of neuromuscular disease research and care for over 70 years. Today there is a robust pipeline of treatments where less than two decades ago there were none. MDA has led the way for the current explosion of scientific information and discoveries pouring out of labs that will lead to new treatments for neuromuscular disease (NMD), providing hope for people with previously untreatable diseases. The path from drug discovery to delivery has been paved by MDA's consistent presence and mission forging innovations in development programs and raising the voice of patients. The point is the pipeline is pretty full in the neurodegenerative space, and we're proud to move the science forward together," said Dr. Woodcock.
MDA Tribute Tour – Washington, D.C. – Schedule
MDA is celebrating the neuromuscular community in Washington, D.C. with partners in healthcare, fundraising, regulatory affairs, grass roots advocacy.
- Thursday, June 1: MDA Care Center visit at Children's National Hospital
- Friday, June 2: MLB Lou Gehrig Day at the Washington Nationals vs Philadelphia Phillies game
- Saturday, June 3: Tribute Awards reception at The National Press Club
About Muscular Dystrophy Association
Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases. For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families. MDA's mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, Twitter, TikTok, and LinkedIn.