Nearly 1,000 boys in the New York Tri-State area have been diagnosed with Duchenne Muscular Dystrophy (DMD) and, until now, had to travel out of the state for comprehensive care. Stony Brook Children's Hospital stepped into the void and is becoming a destination for care for those patients residing in the New York, New Jersey, Connecticut regions and beyond.
18-months in the making, it was ultimately the dedication of the Hope for Javier organization that helped to make this center possible.
"We've been taking Javier, my 12-year-old son, out of state for care since he was diagnosed," said Jennifer Portnoy, Co-Founder, Hope for Javier. "But over the years I've come in contact with a lot of boys in the New York area whose families are not as fortunate as mine and can't afford to travel."
Portnoy wanted to see that change. Through Hope for Javier, she raised more than $600,000 to open a Duchenne Muscular Dystrophy Center at Stony Brook Children's Hospital.
"I want to personally thank Hope for Javier today for their extremely generous commitment, making it possible for Stony Brook to step into this leadership role to become a destination for DMD care for residents of New York, New Jersey, Connecticut and beyond," said Samuel L. Stanley, Jr., MD, President, Stony Brook University, who spoke at the event. "Stony Brook University's strengths in research place us on the leading edge of what is possible. As an academic medical center, we will be able to identify clinical trials as we continue to fight this disease, and your support gives patients and families hope, and it will enable our clinical leadership to provide a level of research, care and support that is unrivaled in the region."
The opening of the Duchenne Muscular Dystrophy Comprehensive Care Center at Stony Brook Children's Hospital marks a new era in the care of patients with muscular dystrophy. The center has modeled its approach to muscular dystrophy on the highest national standards for excellence in care. This model of care is based on precise diagnosis; coordinated, multidisciplinary teamwork; support and advocacy; rapid application of new, evidence-based knowledge; and identification of clinical trials to benefit patients.
"This will be a comprehensive center, including pediatric specialists from neurology, cardiology, pulmonary medicine, gastroenterology, orthopedics, endocrinology, physical, occupational and respiratory therapy, genetics, nutrition, social work, palliative care, child life, and additional support services," said Margaret McGovern, MD, PhD, Professor and Chair, Department of Pediatrics, Stony Brook University School of Medicine, Physician-In-Chief, Stony Brook Children's Hospital, during the ribbon cutting ceremony that took place on Wednesday.
Duchenne Muscular Dystrophy is one of the most common and devastating genetic diseases of childhood, affecting approximately 1 in 5000 boys nationwide. This progressive muscle degeneration leads to loss of ambulation by age 12, loss of upper arm use in the teen years, and then ending a patient's life with heart and respiratory failure in the 20's.
Until recently, boys with DMD usually did not survive beyond their teen years. Today, survival into the early 30s is more common. Studies have proven that access to multidisciplinary care adds on average ten years to DMD patient's life expectancy.
"The difference this program will make in the lives of these patients and families cannot be emphasized enough," said Dr. McGovern. "Our commitment to high-quality, patient-centered care and to groundbreaking medical research will be advanced through our specialized, interdisciplinary clinical team, which is well-equipped to meet the challenges of diagnosing, treating and managing neuromuscular diseases."
Stony Brook Children's is dedicated to improving the care of DMD patients by rapidly bringing evidence-based drug protocols and other advances to these patients-- ultimately better managing and understanding this condition, enabling patients to live a full and long life, and providing family with more time.
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