Health & Wellness

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


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.” 

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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 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

For more information and complete list of locations, visit:

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