Impacting Children with Juvenile Myositis
Updated: Mar 3, 2020
In September of 2017, one week before her 3rd birthday, my daughter was diagnosed with a rare and life-threatening autoimmune disease called Juvenile Myositis. This disease impacts only 3 out of 1 million children in the US each year and causes the immune system to attack healthy skin and muscle tissue in the shoulders, neck, and legs. These attacks cause rashes, inflammation, weakness, and if gone untreated, she wouldn’t be able to swallow or even breathe. When we first saw the rashes on her knuckles, elbows, and knees, we initially just thought she was having an allergic reaction to something we cleaned the floor with. It’s common for toddlers to develop these kinds of things every once and awhile, and so we thought nothing of it. We just applied a variety of over-the-counter creams on the rash — anything else would seem like an overreaction.
It would get a little better, and then worse again for about two weeks. Then we started to notice her forearms looked puffy, kind of like the cartoon character Popeye’s arms, and my wife started getting concerned that the rash was possibly getting infected. She took her to see her pediatrician. Our pediatrician called in the other three doctors of the practice into the exam room, and they huddled together discussing what they were seeing. Then he said that we needed to see a pediatric dermatologist right away, and he got us an appointment with one he recommended for the following day. He had an idea what might be going on, but he wanted to confirm his suspicions with the dermatologist before he made the final call.
Well, one look from the dermatologist, and they diagnosed Ryan with JM. They said to make an appointment with a pediatric rheumatologist right away and to stay off the internet.
“Do NOT Google this disease, because it will scare the heck out of you” they said…
Of course, as a parent, you can’t help but research everything related to your child’s disease. By the time my wife got to her car, she had already pulled up numerous articles on Google about JM. Of course, as with many things, the most sensational stories are the top articles that come up when searching for a rare disease. They definitely did scare the heck out of us, but to be honest, we were still in a kind of denial. Ryan had a rash — yes, that was obvious — but she wasn’t showing muscle weakness, and that was a huge symptom of JM. We held onto the hope that the doctors were wrong about their diagnosis and prayed that a trip to the rheumatologist would prove exactly that.
The following day, my daughter could no longer walk up the stairs in our house, run down the hallway, or even lift her arms above her head. Our hope was crushed. The muscle weakness hit her quickly and hard, and our denial was quickly erased and replaced with the hard reality that this nightmare was real. Our daughter had JM and required immediate treatment. Three days later, we were meeting with the rheumatologist at Hackensack University Medical Center to learn how best to treat Ryan and this terrible disease. This was the beginning of some extremely strenuous years for our family. The following morning we were back at the hospital undergoing her first of 23 rounds of intravenous infusions of IVIG, along with her weekly shots of methotrexate, and daily high doses of Prednisone. The Prednisone continued for 10 months, the IVIG infusions for 23 months, and the Methotrexate shots continue to this day…
During this treatment period, the steroids caused her to bloat and she went from 30 pounds to 45 pounds within three months. The infusions at the hospital were 12-14 hour long days, including the hour and a half drive to home and the 9+ hours hooked up to the IV. She was poked and prodded multiple times each round as the nurses tried to hit a vein for blood work in her little 3 year old arms. She would scream and cry and was terrified each month when we would wake her up at 5:00 am for the drive to the hospital. Within 48 hours after her infusions, she would typically get very sick, with strong headaches and vomiting as the medication worked its way into her body. Several times, she landed in the ER suffering from dehydration. Even now, two and a half years after her diagnosis, she is still scared and cries once per week when my wife and I walk into her bedroom to give her weekly shot. Although the medications are outdated and have many side effects, they have put her disease into medical remission. Once her weekly shots are through, we’re hoping that this coming summer, she will be in full remission. But as with all autoimmune diseases, there is no magic cure. She will live with this disease in her body for her entire life, and there is always a possibility that it will flare up again.
Our biggest fear is that if she does flare up again, she will have to start at square one, back on high doses of Prednisone with all the nasty side effects that come along with it. They made her miserable and sick. Unfortunately, due to the rarity of her disease, there is no financial incentive for companies to develop safer drugs to treat JM. The profitability is just not there given that so few need to be treated. Thankfully, there’s one organization that’s dedicated to funding research and clinical trials for safer medications to treat kids with JM: The Cure JM Foundation.
In 2003, Cure JM was started by a couple in California whose grandson had JM. Their first fundraiser was a humble lemonade stand. Now, Cure JM has grown to a multimillion dollar foundation that funds research with world-class scientists and organizations such as Children’s Hospital of Philadelphia (CHOP) and the National Center for Advancing Translational Sciences (NCATS) at the National Institute of Health. They study genomics to understand the origins of the disease and test the efficacy of off-label uses of other medications for other diseases to see if they will also effectively treat JM, with minimal side effects. Every day they are dedicated to pushing this research forward, and there are several promising trials underway that could make JM a manageable disease for our children. One of their founders, Shari Hume, is excited about the future: “When we started this organization we were hopeful, but uncertain we could find a cure for JM in our children’s lifetime. Now, based upon working with the world’s top researchers, raising awareness, and building an amazing community of dedicated families, I’m certain we WILL find a cure.”
Even though JM is a rare disease, we should do everything in our power to aid this effort by spreading awareness and increase funding. MPC Builders is pledging to donate 10% of the profit from each of our projects to the Cure JM Foundation. These donations will be made in honor of our clients when they choose to award MPC a new project as a way of saying “thank you for your business.” We want to tie the success of our business to a higher cause because we believe that all great companies not only manage their core business well but also contribute to the greater good of humanity. By tying our success to the success of The Cure JM Foundation, every day we will show up to work, motivated to succeed and ready to give it our all each and every day, because we’re not just building a project for our clients… we’re building a better future for my daughter and other rare children like her around the world. Please join us in this fight for safer treatments, and one day, a cure for Juvenile Myositis.
Please consider making a donation to "Ascent 2 A Cure" for JM today! Click Here