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Goryeb Children’s Hospital Designated Nation’s First Pediatric Center of Excellence for Charcot-Marie-Tooth Disease by the Hereditary Neuropathy Foundation

November 14, 2018

Designation Connects Families to Promising Clinical Trials for Disease with No Current Treatment 

November 14, 2018, Morristown, NJ – Atlantic Health System’s Goryeb Children’s Hospital today announced it is the first hospital in the country to be designated as a Pediatric Center of Excellence for Charcot-Marie-Tooth disease (CMT) by the Hereditary Neuropathy Foundation.

“At present, there is no drug therapy for patients with CMT, which can be worrying and frustrating for CMT patients and their families,” said Jahannaz Dastgir, DO, medical director of the Pediatric Neuromuscular Medicine Program at Goryeb Children’s Hospital. “Our new Pediatric Center of Excellence designation will allow them access to promising clinical trials assessing new drug therapy options, and a specialty team that will work with them to manage the condition while these therapies are being researched. This is an area in major need of attention, and it is imperative to work toward the goal of therapeutics.”

In addition to clinical trial access, the designation enables patients to be added to national and international registries and access Goryeb Children’s Hospital’s nationally recognized multidisciplinary (multi-specialist) team designed to assess treat neurological and neuromuscular disorders. For patients with CMT, the team is comprised of a pediatric neurologist, physiatrist, orthopedic surgeon, pulmonologist, physical and occupational therapists and an orthotist.

“Goryeb Children’s Hospital is a well-established children’s hospital that uses the latest advancements and science to treat and provide options for children with inherited neurological disorders,” said Allison Moore, CEO and Founder of the Hereditary Neuropathy Foundation. “Their established program, world-class medical team, and capacity for research made it a strong choice as our first Pediatric Center of Excellence for Charcot-Marie-Tooth disease.”

“Adding Dr. Dastgir to our team has expanded treatment options for children with nerve and muscle disorders,” said Bernard Maria, MD, Chief of Neurosciences, Goryeb Children’s Hospital. “Innovative treatments are now available that we could not even dream about 10 years ago.”

CMT is one of the most common inherited neurological disorders, affecting approximately one in 2,500 people in the United States, and affects the peripheral nervous system. CMT is characterized by progressive loss of nerve function (sensation and motor) and subsequently muscle tissue across various parts of the body, and is most often diagnosed in the early adolescent years.

Patients with CMT can experience weakness of the foot and lower leg muscles, frequent tripping or falls, foot and leg deformities, difficulty with fine motor skills in the fingers, hands, wrists, feet, and tongue and respiratory weakness. Patients are prescribed physical therapy, foot and ankle braces or moderate activity to support their physical limitations.

Dr. Dastgir has led or participated in a number of clinical trials for pediatric patients with neurological and neuromuscular disorders and diseases including pivotal trials for spinal muscular atrophy and Duchenne muscular dystrophy that led to the United States Food and Drug Administration (FDA) approvals of the only drug treatment options. While a clinical research fellow, she was also involved with the development of a clinical protocol for the first spinal gene therapy trial for Giant Axonal Neuropathy, currently underway at the National Institutes of Health.

Specialty services for children with neurological and neuromuscular disorders and diseases at Goryeb Children’s Hospital include services for:

For more information on Atlantic Health System, call 973-971-5700.

About Atlantic Health System

Atlantic Health System, headquartered in Morristown, N.J., is an integrated health care delivery system powered by a workforce of over 16,500 team members and 4,800 affiliated physicians dedicated to building healthier communities. The system serves a population of 5 million, with over 400 sites of care, including six hospitals: Morristown Medical Center, Overlook Medical Center, Newton Medical Center, Chilton Medical Center, Hackettstown Medical Center and Goryeb Children’s Hospital.

Atlantic Health System provides care for the full continuum of health care needs including 11 urgent care centers, Atlantic Rehabilitation and Atlantic Home Care and Hospice. Facilitating the connection between these services on both land and air is the transportation fleet of Atlantic Mobile Health.

In addition to the employed workforce, Atlantic Alliance, a Clinically Integrated Network, represents more than 2,500 health care providers throughout northern and central NJ. Atlantic Health System leads the Healthcare Transformation Consortium, a partnership of 6 regional hospitals and health systems dedicated to improving access and affordability, and is a founding member of both the PIER Consortium – Partners in Innovation, Education, and Research – a streamlined clinical trial system that will expand access to groundbreaking research across 6 health systems in New Jersey and Pennsylvania and AllSpire Health Partners, a consortium of 5 leading health care organizations dedicated to serving patients, families and communities in New Jersey, New York, Maryland and Pennsylvania.