Evelyn Gonzalez has a need for speed.
Her favorite ride at Disneyland is Big Thunder Railroad.
She loves to pop wheelies on her wheelchair and play soccer and even go ziplining on it.
She’s also a huge fan of the Rollettes, an all-female wheelchair dance group based in Los Angeles.
Evelyn’s mother, Lesley, recalls getting a call from school.
“Can she slow down?” a teacher asked of the vivacious third grader, who also loves to perform on stage.
Now, Evelyn has raced to a first at Rady Children’s Health Orange County. She’s the hospital’s first patient, and among the first children in the region, to receive a high dose of the medication Spinraza (nusinersen) to treat her condition, spinal muscular atrophy (SMA).
Pediatric neurologist Dr. Julian Thomas administered the historic dose on Oct. 2, 2026, in the Outpatient Infusion Clinic in the Southwest Tower. The Food and Drug Administration approved the more concentrated Spinraza regimen this March.
Life-saving medication
By optimizing the dosing during the one-time loading phase and every four months after that for maintenance, Dr. Thomas and other neurologists have the opportunity to deliver even greater and more sustained benefits to patients like Evelyn.
Spinraza was first used in human clinical trials in 2011 and received its first U.S. FDA approval in December 2016. Previously, there was no treatment for SMA, a rare genetic neuromuscular disease that causes progressive muscle weakness and deterioration due to the loss of motor neurons in the spinal cord. SMA is the leading fatal disease among infants, and Spinraza has been credited with saving the lives of patients around the world.
Evelyn was diagnosed with SMA just after she turned 1 and has been in and out of the hospital most of her life since then. She first received Spinraza, manufactured by Biogen, in 2020. Lesley and her husband, Anthony, who have a healthy son, Diego, 10, are hopeful the higher-dose regimen will yield even greater benefits.
‘You’re amazing!’
On the day of her infusion, Evelyn was accompanied by her mother and her canine companion, Maren, a 3-year-old pure-bread yellow labrador. Evelyn got Maren a little over a year ago to help her with such tasks as picking up dropped items and plucking clothes out of the hamper to help her do laundry.
Wearing a T-shirt with the phrase “Disabled Isn’t a Bad Word” and sparkly silver sneakers, Evelyn busied herself her a game on her tablet as her care team prepared for the injection.
For years, Evelyn received Spinraza through a lumbar injection. A port inserted behind her right ear last year has made the experience less painful and much quicker. Neurosurgeon emeritus Dr. Michael Muhonen designed the device, highlighting the Neuroscience Institute‘s record of research and innovation.
Dana Moran, assistant nurse manager, Oncology Clinic, stopped by to greet Evelyn and her mother as Child Life specialist Ashley McNamara crouched down beside Evelyn’s bed to play with her as Dr. Thomas prepared the injection.
Nurse Erica Crawford told Evelyn: “You’re amazing! You know that?”
The care team handed Evelyn a vibrating gadget, Buzzy the ladybug, to distract her. An example of how the Neuroscience Institute uses leading-edge technology in a child-centered way, the Buzzy Bee uses the Gate Control Theory of Pain to block sharp injection and IV pain naturally.
Dr. Thomas numbed her with cream before inserting the needle to directly inject the medication into her cervical spine.
The injection, followed by a flush of water to clear out the line, took a minute or so.
“Great, my friend,” Dr. Thomas told Evelyn, who took the procedure like a champion. “Fantastic job.”
Ambitious career plans
In addition to her athletic and artistic endeavors that have includes playing Elsa from “Frozen” on stage, Evelyn, joined by her mother, regularly visits parks to rate them on accessibility. She recently helped get a sidewalk installed to improve access to tennis courts.
Connected to this effort, the Ms. Wheelchair California Leadership Institute crowned Evelyn Mini Miss Wheelchair California 2026 (Adaptive Play).
Evelyn is looking forward to celebrating her 9th birthday at Legoland in November.
Asked what she dreams of becoming when she grows up, Evelyn, true to form, wants a high-energy career:
“A firefighter ballerina doctor.”
Refer to a CHOC Pediatric Neurosurgery Specialist
Referrals can be placed online through the eCeptionist Referral Portal.
