Tucson Boy among First to Receive FDA- Approved Gene Therapy Treatment for Muscular Dystrophy
Seven-year-old Hudson Sanford now has a chance to live out a normal childhood thanks to an FDA-approved gene therapy treatment he received for Duchenne, a rare type of muscular dystrophy.
But, it was a treatment that almost didn’t happen, since it was only approved for children ages four and five years old, and Hudson was just weeks away from turning six.
The Diagnosis
The journey first began when Hudson’s preschool teacher noticed that he wasn’t meeting some milestones like his peers.
“He was using his hands to get off the floor, running a little slower, and wasn’t jumping,” said Melanie Sanford, Hudson’s mother.
After appointments with a neurologist, genetic testing results came back indicating Duchenne—a rare, genetic disease ultimately leading to progressive muscle damage and weakness.
Duchenne typically affects males, and muscle weakness becomes increasingly noticeable between two and five years old. Most children living with Duchenne use a wheelchair beginning around age 13.
“It was really devastating for us,” said Melanie. “We did not have a great first experience and were told there was not much we could do.”
Refusing to accept the discouraging prognosis for their only child, Melanie kept digging for options, eventually stumbling upon gene therapy, which was just coming through clinical trials.
Hope for Hudson
At the time, since the gene therapy treatment for Duchenne was new, it was only available to children ages four and five years old.
“It was so amazing to read about the gene therapy for Duchenne since there’s no other treatment besides steroids,” said Melanie. “But I still wasn’t very hopeful because Hudson was about to turn six.”
Acting quickly, the Sanfords were put in contact with Dr. Diana Castro and the CureDuchenne Clinic in Texas, and just a few days later, had their first appointment.
Although the Sanfords had some hesitations, they ultimately decided to try and go forth with seeking treatment.
“We knew the alternative and what the future would have looked like,” said Melanie. “This would give him the best future possible so we knew it was the right decision.”
After a series of preliminary tests and an appeal after their insurance initially denied it, on August 18th, 2023, Hudson was able to receive the gene therapy infusion, just three days shy of his sixth birthday.
The one-time treatment given through an IV infusion treats the underlying cause of Duchenne by delivering a shortened dystrophin gene to the skeletal muscle.
Melanie said the overall process from start to finish took just a few hours, but the results are having long-lasting impacts.
“He is not even the same child,” said Melanie. “He runs around, jumps, runs up the stairs, he’s even on a bowling league. Around his age, kids usually start to decline, but he’s improved and gained strength, and we’re so happy with everything he’s able to do.”
As Hudson now gets the chance to carry out all the normal day-to-day activities of a seven year old, the Sanfords are feeling grateful for their experience and hope other Duchenne children and their families remain optimistic.
“I want others to know that if they do get this diagnosis it can be devastating, but there are so many people who want to help,” said Melanie. “The future is definitely bright and I feel very thankful for the people who have helped us.”
For more information on Duchenne visit duchenne.com
