How new SMA treatments are turning hope into reality
Together, our community is igniting miracles
Last month, the biotechnology company Scholar Rock submitted an application to the U.S. Food and Drug Administration requesting approval of an experimental add-on therapy to treat spinal muscular atrophy (SMA). The investigational drug apitegromab is intended to target a protein in the bloodstream that inhibits muscle growth with the hope that SMA patients can rebuild strength.
Lately, I’ve been scouring the internet for success stories from the apitegromab clinical trials. Although I don’t know if I’m the ideal candidate for this treatment, as it’s only been tested in patients ages 2 to 21 and I’m in my 30s, this drug sounds so promising for children and young adults. It’s incredible to witness.
With three SMA treatments already available and one hopefully to be approved later this year, the SMA community is truly on the precipice of a brighter future.
‘I believe in miracles’
Years ago, life with this disease was an entirely different story. There were no treatments or clinical trials. Doctors were limited in knowledge and had minimal experience with patients living with SMA. Hope was the guiding light in a seemingly dark world.
When I was diagnosed in April 1991, my parents clung tightly to that feeling. I can recall stories they’ve shared with me about my diagnosis day and how profoundly sad they were. My official diagnosis was Werdnig-Hoffmann disease, or SMA type 1, and was limited to two sentences in an old medical book my pediatrician had in his office. There wasn’t much guidance he could give my parents. The internet wasn’t widely available yet, either. All he knew was that my circumstances were bleak, but he left my parents with four simple yet profound words that still resonate today:
“I believe in miracles.”
In their darkest hour, someone gave my parents a breadcrumb of hope. Life as they knew it was about to change for good. There were no road maps or playbooks on how to navigate life with SMA. They had no experience with this disease or anyone to turn to for advice. Instead, they had an abundance of love, support from family, and faith that miracles can happen.
Today, at 34 years old, I understand what my pediatrician meant when he spoke those words to my parents. There have been little miracles everywhere these last few decades, not just in my life but also in the entire SMA community. The researchers who work tirelessly to find treatments have given new hope to families. The people and organizations who generously donate their time and money to help fund these efforts have forged a new path forward. The doctors, nurses, families, and advocates have been the heartbeat of this community that keeps moving forward. All of us, together, have ignited glimmers of miracles along the way.
I don’t know if I’ll be eligible for apitegromab if and when it becomes available. Because I’m considered an “older” adult with SMA, my muscles may be too weakened to reap the benefits of this drug. But what I do know is that there has never been a better time in history for SMA. The progress and advancements made in recent decades are nothing short of miraculous. How lucky am I to be witnessing it?
Note: SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of SMA News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to spinal muscular atrophy.
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