MARQUETTE — The FDA recently approved a breakthrough treatment for people who suffer from spinal muscular atrophy also known as SMA. ABC 10’s Melanie Palmer spoke with an Ishpeming native who suffers from the disease and asked her reaction to this groundbreaking treatment.
You won’t live past the age of 2 is what Molly McGlone’s family was told after she was diagnosed with spinal muscular atrophy at the age of 10 months. Molly has far exceeded doctor’s expectations as she is now a college student pursuing a degree in Pre-med. There was no approved treatment for this disease until December 23rd of this year, when the FDA approved its first ever therapy for SMA.
After Molly heard about this breakthrough treatment, she was beyond excited.
“I was just scrolling through Facebook and was like ‘Oh, my God’ and I thought my heart was going to jump right out of my chest and then I texted my friends and I cried a lot,” said NMU student with SMA, Molly McGlone.
According to CureSMA.org, this disease affects 1 in every 10,000 babies and 1 in every 50 Americans is a genetic carrier.
This genetic disease affects the motor nerve cells in the spinal cord, taking away patient’s abilities to walk, move, eat, and at times even breathe. The recently approved treatment, Spinraza is a synthetic genetic material that binds RNA. In total, it would fix splicing errors in the affected genes, this of which could assist with muscle movements.
Knowing this treatment is now available, gives Molly something important to hold on to.
“Hope that it can benefit our lives and I want to say adults who do get treatment, they might not benefit greatly but even getting a little muscle back, that would be nice,” said McGlone.
Molly also wanted to emphasize how grateful she is for all the kids and babies who get diagnosed that now have a treatment option. With plans of receiving treatment in the near future, there is one goal Molly has in mind.
“If I get enough motor function, my first goal would to be to learn violin. I would love that, I’ve always wanted a violin,” said McGlone.
Molly finds her escape through music and is passionate about creating it herself. Although there have been battles in Molly’s past, she wouldn’t change anything about herself. Having an outstanding support group is what also keeps Molly optimistic.
“I got great family and great friends. Shout out to my friends who pick my head up when it falls over,” said McGlone.
Molly’s plans on going into pre-med transpired from the time she spent in the hospital at a young age. Being a compassionate, caring doctor that will give hope to children is what she aspires to be in the future.