NORFOLK, Va. – A 3-month-old patient at Children’s Hospital of The King’s Daughters was the first child in Virginia, and among the first in the country, to receive a new gene therapy drug since the Food and Drug Administration approved it to treat a disease that’s the top genetic killer of babies.

Pediatric neurologist Crystal Proud diagnosed Kaeli Price of North Carolina with spinal muscular atrophy in May when she was 8 weeks old. SMA is a progressive neuromuscular disease that gradually wears down muscles to the point where children are unable to walk or breathe easily. Children with the most severe forms need ventilators to breathe and usually die in childhood.

But on May 24, a week after Kaeli was diagnosed, the federal Food and Drug Administration approved a new gene therapy drug, Zolgensma, for treatment of SMA. Dr. Proud arranged for Kaeli to receive the drug on June 20 at CHKD just weeks after the first baby in the country received the newly approved treatment in Florida.

“My hope for Kaeli is that she’ll be able to sit and stand and walk and live a fruitful life,” Dr. Proud said. “It’s a life-changing drug that’s given us new hope and optimism we didn’t have before.”

This is the first gene therapy treatment for a neuromuscular disease, and it’s made headlines across the globe because drug company Novartis priced it at $2.1 million a patient, setting a world price record.

Kaeli’s parents, Kelli and Brandon Price of Belvidere, N.C., feel fortunate that the drug, which is for children 2 years of age and younger, became available when it did, and that their insurance company agreed to cover it. “It’s hard to put into words how we feel,” said Kelli Price. “We feel like the timing was all God’s doing.”

There was no approved medical treatment for SMA until 2016, when the FDA approved a drug called Spinraza, which allows a backup gene to produce more of a protein needed for motor neuron development. Dr. Proud, a pediatric neuromuscular neurologist, began administering Spinraza in 2017 and now treats about 30 patients with the injections into the spinal canal.

Zolgensma, though, is a one-time infusion that uses a virus to provide a normal copy of a gene that is missing or mutated in children with SMA. The drug could ultimately be less expensive and less trouble for patients than Spinraza, which must be administered repeatedly. Dr. Proud treated a 5-month-old patient with SMA at CHKD with the gene therapy in February when the drug was still in the clinical trial phase. The baby was authorized to receive the drug by the FDA under the compassionate use, or expanded access, program, a way patients with an immediately life-threatening condition can gain access to an investigational drug outside a clinical trial. That baby, who lives in another part of the state, is doing well.

Patients in clinical trials of the gene therapy showed improvement in the two years following treatment — they could breathe without a ventilator and some were able to talk, sit and even stand on their own, developmental milestones that many patients with SMA never reach. Studies are still tracking its long-term effectiveness.

Spinal muscular atrophy affects one in every 10,000 births. Dr. Proud has been involved in a statewide steering committee to recommend newborn screening for SMA in Virginia, so early intervention can provide the best development opportunities for these children.

For more information and to view a video about the infusion, visit CHKD's Spinal Muscular Atrophy Center webpage.

Left: Kaeli Price sits in the lap of her mother, Kelli Price, as she receives an infusion of a gene therapy treatment, Zolgensma.
Right: CHKD Neurologist Crystal Proud checks on Kaeli Price just before she begins the gene therapy infusion expected to extend her life.

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CHKD is the only freestanding children’s hospital in Virginia and serves the medical and surgical needs of children throughout greater Hampton Roads, the Eastern Shore of Virginia, and northeastern North Carolina. The not-for-profit CHKD Health System operates primary care pediatric practices, surgical practices, multi-service health centers, urgent care centers, and satellite offices throughout its service region. Learn more at CHKD.org.

HAMPTON ROADS, Va. – Hampton Roads Walmart stores and Sam’s Clubs will be raising money now through July 7 to help kids treated at CHKD. Customers and members can donate $1 or more at the checkout lane or self-checkout at any Walmart Store or Sam’s Club in Hampton Roads.

The annual campaign for Children’s Miracle Network Hospitals raises money for 170 children’s hospitals in the United States and Canada. To date, Walmart and Sam’s Club associates, members, and customers have raised more than $1 billion for the network’s hospitals, including $11.7 million for CHKD.

“This campaign is very important to our associates and customers, and every year they do a great job raising funds and awareness for children in the area,” said Brent Rains, Walmart Regional Manager in Virginia. “We are always hoping to make a difference in the community and we are excited to kick off this year’s campaign with the goal to help kids in need.”

Thirty-nine Walmart Supercenters, Neighborhood Markets, and Sam’s Clubs in Hampton Roads will participate, with a goal of raising $550,000. Help them reach their goal and fund critical treatment, equipment, research, and charitable care for CHKD kids at the same time!

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CHKD is the only freestanding children’s hospital in Virginia and serves the medical and surgical needs of children throughout greater Hampton Roads, the Eastern Shore of Virginia, and northeastern North Carolina. The not-for-profit CHKD Health System operates primary care pediatric practices, surgical practices, multi-service health centers, urgent care centers, and satellite offices throughout its service region. Learn more at CHKD.org.

HAMPTON ROADS, Va. – CHKD will be kicking off Mental Health Awareness Month by lighting its buildings green in May, and hosting a month of events to promote the mental health of children.

One of the highlights will be a screening of “Angst: Raising Awareness Around Anxiety,” an inspirational documentary that features children and young adults speaking about anxiety. CHKD is presenting the screening in partnership with the Cameron K. Gallagher Foundation, a Richmond-based nonprofit dedicated to erasing stigmas, raising awareness, and providing mental health education. Cameron Gallagher, who struggled with these issues, lost her life due to an undiagnosed heart condition just after she crossed the finish line of the 2014 Shamrock Half Marathon.

A panel discussion with CHKD experts and special guests will follow the screening. Media representatives are invited to interview these experts on anxiety to advance the event.

The IndieFlix film about anxiety – the most common mental condition in the country – features an interview with Olympic swimmer Michael Phelps. He shares his story of being bullied and depressed, which led to severe anxiety. The winner of 28 Olympic medals encourages others with anxiety to talk about it and reach out for help.

A popular and well-received documentary, the Angst screening and discussion will be May 2, from 6:30 to 8:30 p.m., in the Sparks Science Center at Cape Henry Collegiate, 1320 Mill Dam Road in Virginia Beach. The event, suitable for children 10 and older, is free, but space is limited. Visit CHKD.org/Angst to register. Representatives from the CKG Foundation will also attend.

• CHKD also will be lighting buildings green the month of May, in partnership with other organizations across the country, including the National Alliance on Mental Illness Coastal Virginia, which will host a lighting ceremony on May 1, at 11:30 a.m., at Town Center Square fountain in Virginia Beach. Besides the main hospital, CHKD will light CHKD Health Centers and Urgent Cares at these locations: 1924 Landstown Centre Way, Virginia Beach; 817 Volvo Parkway, Chesapeake; 3960 Virginia Beach Boulevard, Virginia Beach; 680 Oyster Point Road, Newport News.
• This year’s CHKD RunWalk for The Kids on May 11 will be dedicated to mental health, with proceeds benefiting CHKD’s mental health program. The 14th annual RunWalk will be at the Waterside District in Norfolk and will include an 8K run, a 2-mile walk, and a 1-mile FunRun for kids. To register, visit RunWalkForTheKids.org.
• For a full schedule of events, visit CHKD’s Mental Health Month Calendar of Events.

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CHKD is the only freestanding children’s hospital in Virginia and serves the medical and surgical needs of children throughout greater Hampton Roads, the Eastern Shore of Virginia, and northeastern North Carolina. The not-for-profit CHKD Health System operates primary care pediatric practices, surgical practices, multi-service health centers, urgent care centers, and satellite offices throughout its service region. 

NORFOLK, Va. – The neuroscience unit at CHKD has been accredited as an esteemed Level 3 epilepsy center by the National Association of Epilepsy Centers, making it one of four pediatric facilities in the state to be certified.

The NAEC accredits Level 3 centers for providing a comprehensive team approach to the diagnosis and treatment of epilepsy, including medical, neurological, and psychological aspects. These centers bring together neurologists, neurosurgeons, nurse specialists, EEG technologists, pharmacists, social workers, and others to provide an integrated system of care.

The organization defines four levels of centers, and grants certifications to the two highest, Levels 3 and 4. In Virginia, eight hospitals are certified, with CHKD the only free-standing children’s hospital to achieve that designation.

"CHKD’s epilepsy center brings together highly trained pediatric specialists who work as a team using the most advanced monitoring equipment,” said Dr. Michael Strunc, a CHKD pediatric neurologist who is the medical director of the neuroscience unit.  “We’re honored to have earned this designation that reflects our commitment to children with neurological disorders.”

At CHKD, four video EEG rooms with state-of-the-art diagnostic equipment enable neurologists and epileptologists – neurologists who specialize in epilepsy – to confirm the presence of seizures and to monitor them over time.

The most common treatment is medication, based on the type of seizure, age of the child, side effects, cost, and east of use. The center also offers surgical options. There’s also a treatment called “vagus nerve stimulator,” in which a pacemaker-like device is implanted to stimulate the vagus nerve, which runs from the brain to the torso. The device sends small pulses of energy to the brain to stop seizures. It works without patients even being aware of it most of the time, but if they sense a seizure coming on, they can swipe a magnet over the left chest area to send an extra burst of stimulation.

The unit also has a dietitian who oversees a ketogenic diet program, which is a special type of diet that reduces seizures in some children for whom medication doesn’t work.

In the two years since the neuroscience unit opened, close to 900 patients with seizures have been treated. For more information on epilepsy and treatments at CHKD, visit this website:  CHKD Epilepsy Program.

A 6-year-old patient is evaluated at CHKD's neuroscience unit, which was just accredited as a Level 3 epilepsy center by the American Association of Epilepsy Centers. The designation means the unit provides comprehensive, integrated services for the diagnosis and treatment of epilepsy.

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CHKD is the only freestanding children’s hospital in Virginia and serves the medical and surgical needs of children throughout greater Hampton Roads, the Eastern Shore of Virginia, and northeastern North Carolina. The not-for-profit CHKD Health System operates primary care pediatric practices, surgical practices, multi-service health centers, urgent care centers, and satellite offices throughout its service region.