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Goryeb Children’s Hospital First Hospital in New Jersey to Administer Newly Approved Treatment for Rare Genetic Disease

March 22, 2017, Morristown, NJ – Goryeb Children’s Hospital at Morristown Medical Center, part of Atlantic Health System, was the first hospital in the state to administer nusinersen, a newly approved medication to a patient with spinal muscular atrophy (SMA), a rare and sometimes fatal genetic disease that causes progressive loss of muscle strength and movement.

Because of the demand for the product among the SMA community, nusinersen was approved by the U.S. Food and Drug Administration via Priority Review process on December 23, 2016, and administered to 17-year-old Griffen Kingkiner of Montgomery in early February. The medication is designed to improve motor function and slow its decline.

“When the medication was approved on December 23rd, 2016, at 4:51 PM, the entire SMA community breathed a collective sigh of relief, but it soon became apparent that hospitals were not ready to treat patients,” said Beth Kingkiner, Griffen’s mother. “The team at Morristown Medical Center and Goryeb Children’s Hospital were able to bring all the departments and organizations together and provide this treatment to Griffen. We were prepared to travel to another state for treatment, but knowing this facility was 40 minutes from home was a happy surprise.”  

Dr. Jahannaz Dastgir, medical director of the newly launched Pediatric Neuromuscular Medicine Program at Goryeb Children’s Hospital was an investigator on the clinical trials that helped the medication become approved.

“Helping patients like Griffen is the reason I do the work I do the thought that new medications can actually help save or improve motor skills is astounding to me,” said Dastgir. “I am proud to have played a part in Griffen’s treatment, and know it is supplemented with the comprehensive care team in place at Goryeb Children’s Hospital to assist the needs of our pediatric patients with SMA and other neurological diseases and disorders.”

Griffen will attend college in the fall, and hopes to study medicine or computer science.

“Griffen hasn't been sitting on the sidelines waiting for a treatment he's been living his life,” said Beth. “His academic accomplishments are many but what stands out most to me is the resilient, persistent, driven and focused young man I have had the privilege to watch him become.”

Specialty services for children with neurological and neuromuscular disorders and diseases are available through Atlantic Health System Children’s Health and 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

Learn more and view locations > 

About Spinal Muscular Atrophy
According to Cure SMA advocacy group, SMA is a disease that robs people of physical strength by affecting the motor nerve cells in the spinal cord, taking away the ability to walk, eat, or breathe. It is the number one genetic cause of death for infants.

SMA is caused by a mutation in the survival motor neuron gene 1. In a healthy person, this gene produces a protein that is critical to the function of the nerves that control our muscles. Without it, those nerve cells cannot properly function and eventually die, leading to debilitating and often fatal muscle weakness.

SMA affects approximately 1 in 10,000 babies, and about 1 in every 50 Americans is a genetic carrier. SMA can affect any race or gender.

There are four primary types of SMA I, II, III, and IV based on age of onset and highest physical milestone achieved.

Individuals with SMA have difficulty performing the basic functions of life, like breathing and swallowing. However, SMA does not affect a person’s ability to think, learn, and build relationships with others.

Learn more > 

About Atlantic Health System
Atlantic Health System, headquartered in Morristown, New Jersey, is a leading non-profit health care delivery system in New Jersey. Our network includes Morristown Medical Center in Morristown, NJ; Overlook Medical Center in Summit, NJ; Newton Medical Center in Newton, NJ; Chilton Medical Center in Pompton Plains, NJ; Hackettstown Medical Center in Hackettstown, NJ; and Goryeb Children’s Hospital in Morristown, NJ, as well as Atlantic Rehabilitation, and Atlantic Home Care and Hospice. It also includes its subsidiary, Atlantic Ambulance Corporation. Atlantic Health System comprises 1,747 licensed beds, more than 14,000 employees and more than 4,000 physicians. Atlantic Health System has a medical school affiliation with the Sidney Kimmel Medical College at Thomas Jefferson University; is part of Atlantic Accountable Care Organization, one of the largest ACOs in the nation, and is a member of AllSpire Health Partners.

Morristown Medical Center

100 Madison Avenue
Morristown, NJ 07960
973-971-5000

Overlook Medical Center

99 Beauvoir Avenue
Summit, NJ 07901
908-522-2000

Newton Medical Center

175 High Street
Newton, NJ 07860
973-383-2121

Chilton Medical Center

97 West Parkway
Pompton Plains, NJ 07444
973-831-5000

Hackettstown Medical Center

651 Willow Grove Street
Hackettstown, NJ 07840
908-852-5100

Goryeb Children's Hospital

100 Madison Avenue
Morristown, NJ 07960
973-971-5200


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Atlantic Medical Group

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providers.
1-844-264-2250