Local girl with rare condition meets man behind life-changing drug
A local second grader who’s serving as this year’s national ambassador of the Muscular Dystrophy Association got the chance to meet the man who developed the drug that has changed her life.
“I’m happy and excited,” said Faith Fortenberry, who has spinal muscular atrophy or SMA type 2, which made even simple tasks like brushing her teeth and raising her arms above her head a challenge in the past.
“He” is Dr. Adrian Krainer, a professor at St. Giles Foundation and program chair of cancer and molecular biology at Cold Spring Harbor Laboratory in New York who developed a drug called Spinraza, the first and only FDA approved medication to treat Faith’s condition.
On Thursday Krainer delivered a lecture at Baylor about the drug, which Faith and her family were invited to attend.
“I felt like I was meeting Elvis Presley or the president,” Faith’s mother, Leeann, said.
“I'm flabbergasted that he has taken his time to come here and meet with Baylor and that we get to speak with him.”
Krainer lab developed Spinraza which was approved in December 2016.
After months of back and forth with the family’s insurance provider, which at first balked at the hefty price tag of $750,000 for the first year, Faith received her first injection in May 2017 at Cook Children’s Hospital in Fort Worth.
Just thirty days later, her parents were already sharing with followers the great improvement they’d witnessed.
“THIS GIRL has NEVER been able to reach her arms behind her back AND certainly never clasp them together!!!! Omg!!!!” Leeann posted.
“She also has NEVER been able to move around in tub without tumbling over - usually braces herself with both hands! She's totally sitting unsupported & moving around with MUCH BETTER trunk control & hasn't tumbled over with all this wiggling!! Wowzers! “
Leeann said Spinraza gives not only gives her daughter the chance to break down physical barriers but gives their entire family hope.
“When we first got diagnosed we didn't have any hope,” she said.
“We didn’t know where to go or what to do so the fact that the man who invented the medicine who has dramatically changed her life and saved her life and is allowing her to do things that she wasn’t able to do and prolong her life….just the fact that I’m positive we'll be able to love her a lot longer than we thought we would because of Dr. Krainer and what he's done in his research all these years.”
It’s a sentiment echoed by the doctor who talked with Faith in the lobby of the Baylor science building before addressing the crowd.
“It is a wonderful feeling,” Krainer said.
“I have met many SMA patients and their parents and families but every new patient or family that I meet, it feels like the first time.”
“It's just great to see that the research that we have done have helped people that have had a need for a drug because of a disease that has effected them.”
“Nothing else compares to it.”