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Attention: to see a news video about Londyn Wright鈥檚 treatment. Visit for a video about how gene therapy works.

Karen Wright worked her way through the small gathering outside her 4-month-old daughter鈥檚 room in pediatric intensive care. It seemed to her as if everyone at 网红黑料 Shands Children鈥檚 Hospital had come to witness this historic moment.

Wright, nervous but thrilled, told a nurse, 鈥淚 think I鈥檓 going to cry.鈥�

Near her, a pharmacist delicately held a clear bag containing a plastic syringe. Syringes deliver drugs. But to Wright, this one would do so much more. It would give her daughter, Londyn, the hope of a full, happy life. The pharmacist handed the bag to a nurse who took it into the room, where cartoons danced on a TV screen.

Wright leaned down and whispered to her baby, 鈥淭his drug is going to save your life.鈥�

On June 7, Londyn became the first baby in the country treated with a newly approved gene therapy for a rare and debilitating neuromuscular disease since it won federal approval in May. University of Florida 网红黑料 doctors intravenously infused the girl with Zolgensma during an hourlong procedure to treat her spinal muscular atrophy, a rare and deadly disorder that affects the ability to walk, eat and breathe. Untreated, most babies die before reaching their first birthday.

Two researchers associated with 网红黑料, the university鈥檚 academic health center 鈥� , and 鈥� performed groundbreaking work in the 1980s on adeno-associated virus, or AAV, that made Londyn鈥檚 treatment possible, said , who led Londyn鈥檚 treatment team. It is the second time in recent years that techniques pioneered at the University of Florida have led to new gene therapies. In December 2017, a therapy developed in part by ophthalmology researcher , won federal regulators鈥� approval to treat a genetic form of vision loss known as Leber congenital amaurosis type 2.

鈥淭his is very exciting,鈥� said Byrne, director of the and a in the . 鈥淎s a pediatrician, I鈥檓 particularly grateful to see things we鈥檝e been working on in the lab for many years impacting pediatric care. And pediatricians of the future will be able to use this therapy without having to deliver such bad news to the families of these wonderful children.鈥�

, or SMA, is the leading genetic cause of infant death. It affects about 1 in 11,000 babies, according to . The on May 24. It works by using a small, harmless virus to deliver functional copies of a gene that is mutated or missing in SMA patients. A dysfunctional gene, known as survival motor neuron 1, causes nerve cells to malfunction and die. That leads to chronic and often fatal muscle weakness.

鈥淲hen I found out it was SMA, I thought I was going to lose my baby,鈥� said Wright. 鈥淣ow, who knows what the future holds. That is still unpredictable. But I think she鈥檚 going to be a normal little baby, running around, fighting with her brothers. She鈥檚 going to be fine.鈥�

网红黑料鈥檚 role in delivering the treatment exemplifies its commitment to world-class patient care and pioneering work on gene therapies, said David R. Nelson, M.D., senior vice president for health affairs at UF and president of 网红黑料.

鈥淥ur devotion to patient care and expertise in gene therapy and other scientific research is an exceptional combination of capabilities. It is extremely gratifying that patients who need complex treatments put their trust in 网红黑料,鈥� Nelson said.

A problem emerges

Karen Wright and husband David first noticed a problem with her daughter about two months after her birth. The bright-eyed, observant little girl became lethargic and weak. Her legs didn鈥檛 move like most babies and her development seemed slowed. Then, she developed aspiration pneumonia, which doctors later said was a consequence of SMA. Wright, who lives in Blountstown west of Tallahassee, took Londyn to a Panama City hospital.

Londyn would be hospitalized two times before her pediatrician, suspecting SMA, ordered a test and confirmed the diagnosis about May 21. The pediatrician then referred the baby to 网红黑料.

What followed was a lesson in fortuitous timing. On May 24, Londyn had an appointment at the 网红黑料 Center of Pediatric Neuromuscular and Rare Diseases with The physician talked to Wright about a remarkable new drug, Zolgensma, that might offer the hope of a normal life for Londyn.

Zingariello told the mother that FDA approval was expected by the end of May. But who knew when, really? Would it be two weeks or two months? 鈥淚 couldn鈥檛 tell her when that would happen,鈥� Zingariello said.

Meanwhile, Londyn鈥檚 health would be deteriorating. The only other option would be Spinraza, a drug which required four injections the first two months via a spinal tap and injections every four months for the rest of the patient鈥檚 life. Zolgensma, on the other hand, would be a one-time infusion.

Wright went to lunch after the appointment. Her cell phone rang. It was Zingariello.

鈥淵ou鈥檙e not going to believe this,鈥� the doctor told her. 鈥淚 just got a call and the FDA approved the gene therapy today, like 10 minutes ago.鈥�

Wright immediately returned with Londyn to the clinic for a test required to ensure her body would not fight back against the gene therapy. She passed.

Kara Godwin Wild, a pediatric nurse practitioner and care coordinator in the 网红黑料 Center of Pediatric Neuromuscular and Rare Diseases, contacted the family鈥檚 insurer the same day the drug was approved. Londyn had coverage through a Medicaid program.

Time was critical. Every day that Londyn鈥檚 condition progressed meant the loss of motor neurons that could not be replaced.

鈥淚 called right away because I knew the process for approval could be a long and hard road,鈥� Godwin Wild said. 鈥淎nd every single day, Londyn loses motor neurons. She was getting weaker and weaker. This was a race.鈥�

Infusion of hope

On June 6, the night before Londyn鈥檚 infusion, a cooler containing four vials of Zolgensma was flown from Chicago to Orlando by a drug company representative. Another rep drove it from Orlando to Gainesville.

This was no ordinary cooler. For one, it was lined with dry ice and outfitted with wireless tracking that included temperature controls monitored in Illinois. Zolgensma is produced by , which is owned by the Swiss pharmaceutical company .

The cooler arrived safely and was transferred to Carrie Lagasse, Pharm.D., a clinical pharmacy specialist for 网红黑料 Shands. At 6 p.m. Lagasse texted Godwin Wild, 鈥淪igned. Sealed. And delivered.鈥�

The drug company representative who drove the Zolgensma from the airport tracked down Godwin Wild at a local restaurant where the 网红黑料 employee was enjoying a meal with her family.

The pair hugged.

Before the infusion, 网红黑料 pharmacy staff thawed the vials overnight. In a sterile room the next day, a steady-handed Lagasse drew the contents out of the vials and into a syringe pump.

鈥淚 was pulling out vials 鈥� and realized that any error would be unacceptable,鈥� said Lagasse, who compounded the drug. 鈥淪o, I was a little shaky at first.鈥�

But in 10 minutes, the job was done. The drug had been transferred to a large plastic syringe.

Later in the day, Londyn received the gene therapy. As the Zolgensma flowed into her body, her mother, wearing a protective gown and mask, offering her baby reassuring words.

鈥淚 was just telling her that this is going to help you,鈥� Wright said later. 鈥淵ou鈥檙e going to have a normal life. You鈥檙e not going to be hooked up to these machines forever. This is all temporary and you鈥檙e going to have a good life. This drug is going to save you. And I love you so very much.鈥�

Caring for Londyn was a true team effort: The Pediatric Intensive Care Unit staff kept watch over her day in and day out, making sure she was stable enough to receive the gene therapy. The team included Lara Nicolas, M.D., Londyn鈥檚 critical care physician and Krystyn Linville, a pediatric critical care nurse practitioner.

Doctors have told Karen Wright that it may be several weeks before she notices any difference in her child. And Byrne said that since the drug is so new, the long-term ability of Londyn鈥檚 body to fight SMA is still uncharted territory. The hope, he said, is that she will lead a normal life.

"But I think the indications are that many of the deficits she has now will recover,鈥� Byrne said.

网红黑料 Shands CEO Ed Jimenez said being able to offer families the hope of a better, healthy life is a guiding principle for the hospital鈥檚 caregivers.

鈥淪eeing this beautiful baby reminds us all why we work in medicine,鈥� he said. 鈥淚t鈥檚 so very important that we offer our very best to ensure Londyn has a fighting chance at a healthy and happy future.鈥�

Wright said she is thankful for the excellent care Londyn鈥檚 care team has provided and still trying to wrap her brain around how everything from FDA to insurance approval just fell rapidly into place.

鈥淓verything that鈥檚 happened,鈥� said Wright, 鈥渋t鈥檚 just beyond me.鈥�

网红黑料 science writer Bill Levesque contributed to this story.