MORRISTOWN, NJ - Atlantic Health System’s Goryeb Children’s Hospital has been designated as a Pediatric Center of Excellence for Charcot-Marie-Tooth disease (CMT) by the Hereditary Neuropathy Foundation -- the first hospital to earn that designation in the country.

“At present, there is no drug therapy for patients with CMT, which can be worrying and frustrating for CMT patients and their families,” said Dr. Jahannaz Dastgir, 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.

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“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 Dr. Bernard Maria, 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:

  • Autism and development – Goryeb Autism and Development Center (GADC)
  • Brain and spinal tumors – Goryeb Neuro-Oncology Program (GNOP)
  • Concussion – Goryeb Concussion Program (GCP)
  • Epilepsy – Goryeb Epilepsy Program (GEP)
  • Headache and Migraine – Goryeb Headache Program (GHP)
  • Muscle and Nerve diseases – Goryeb Neuromuscular Program (GNP)
  • All general neurology problems

 For more information on Atlantic Health System visit atlantichealth.org or call 973-971-5700.