COLUMBUS, Ohio — Families in Columbus and nationwide are praising a new FDA approved gene therapy for Spinal Muscular Atrophy or SMA. Researchers from Nationwide Children's Hospital and The Ohio State University’s College of Medicine were leaders in the clinical trials. But for some researchers, the drive to find a cure was all because of a girl named Madison.
Madison Reed is just like any other girl, she loves movies, concerts, and celebrities. But what's unique about her is her ability to defy the odds.
Madison was diagnosed with a rare and debilitating disease called Spinal Muscular Atrophy or SMA when she was a baby. Most babies with the disease die or become reliant on respirators and feeding tubes.
"We were told just to take her home and make her comfortable and just wait it out,” said Madison’s mother, Annette Reed.
Doctors told her mom, Annette Reed, she probably wouldn't live more than 18 months.
"It was like we were living in a nightmare and for several months I was in a daze. I didn't know what to do," said Reed.
Madison is now 23.
Early on her parents Annette and Carl Reed were determined to help find to a cure. That's when they started the foundation, Miracle for Madison and Friends.
The money raised through the foundation went on to fund some pre-clinical work done at Nationwide Children's Hospital and The Ohio State University College of Medicine. That work eventually led to the one-time treatment for SMA called Zolgensma.
"If it's given early enough and right after diagnosis, there are kids that are standing and walking, talking, acting normal,” said Reed.
"I'm very proud that we've been able to go from identifying the gene to actual therapies that are actually in practice and are actually being effective,” said Arthur Burghes, Ohio State University College of Medicine researcher and professor.
Unfortunately, the treatment won't help Madison because it can only treat children under the age of two. However, there is a treatment in the works right now that could one day help Madison.
"It's been definitely a miracle. And that's what our aim was for a miracle for Madison and friends,” said Reed.