Hope for Clara Faye; Journey with SMA
Updated: My husband and I originally started this gofundme campaign to pay for the most expensive treatment in the world for our daughter before her second birthday. In the three days before Clara’s birthday, we could not have predicted how far her story would spread and how many people would want to help. Before that Friday morning when insurance finally gave us the news we had desperately been waiting to hear, we weren’t sure what her future was going to look like with treatments. A lot of prayers and persistence helped make Zolgensma happen for our little girl. Any further motor neuron loss attributed to her disease has been arrested, but her journey is not over.
We will be continuing physical therapy twice a week to make sure that she is building strength with the Zolgensma helping her to retain any gains she makes. Stanford made it known to us that, oftentimes, depending on the efficacy of the treatment and what Clara is able to do, more treatments may be necessary in the future, as well as equipment, like gait trainers, motorized chairs, etc. We want nothing more than to see our little girl walk and feel the earth beneath her feet. The future is just never guaranteed and especially with SMA, Clara’s story has the potential to go many different ways. Of course, we always hope and pray for the best and are more than certain that God has his angels watching over her. We know who holds tomorrow, but we don’t know what it might bring.
For everyone who donated and wants to know what the money accrued will be used for, everything is still all for Clara and her road to recovery and living with SMA. Every last penny will be going towards any future treatments, combination therapies, equipment, medications, physical therapies and travel expenses to and from Stanford. She will need to be seen and monitored by the neuromuscular clinic once every week and will also continue being seen by cardiology, fluoroscopy and pulmonary. As studies continue to look at SMA and how it affects the body, more and more is uncovered. We were given ten days to make Zolgensma happen. We do not ever want to be as unprepared for that kind of timeline ever again. Anything that comes our daughter’s way that can make her life better, we want to be able to do, no questions asked.
We are working on getting better coverage for our daughter’s needs, but we have been told that the waiting list is almost a year long in our state, especially after 2020 and the impact of the pandemic. While we wait to find additional financial help for our girl, we will be relying on what we were able to raise in the past few days. A single motorized wheel chair costs upwards of $100,000. We are hoping that Clara will really take to physical therapy and is able to work towards some standing and, God willing, walking, but whatever she feels she is able to do, we want to be able to make it happen. I know we started all of this with the hope of reaching the impossibly high goal of $2.2 million. The good news is, Clara will never need anything that expensive ever again. Her future is still full of medical expenses, most of them unknowns to us.
Every dollar that was raised for Clara is still all for her health and benefit and will give us some breathing room while we navigate what she will need next. Again, we could not have had this peace of mind without all of the support, suggestions, donations and prayers given for Clara these past few days and continue to be so grateful and touched by the continued support. Thank you for giving all three of us hope.
Original: In the summer of 2020, myself and my husband noticed that our then 1.5 year old daughter was not meeting her gross motor milestones, like crawling or walking. Over the next few months, we met with Early Intervention here in Nevada, a child neurologist and various specialists to figure out why Clara had such low muscle tone and motor control. After an EKG, EEG, various metabolic panels, blood tests and an MRI, we submitted genetic testing at the end of February 2021. On March 23rd, we received a world-shaking diagnosis of Spinal Muscular Atrophy, an incredibly rare and deadly genetic neuromuscular disease caused by the progressive loss of the SMN gene which controls all muscle movement, including things like walking, crawling, sitting, swallowing, even breathing. By the grace of God, we were able to see the neuromuscular specialists at Stanford the following week on Wednesday the 31st. They confirmed that Clara has type 2 SMA, which means the most she can do on her own is sit. Without treatment, she will grow weaker and her muscles will continue to waste (or atrophy) over time. Thankfully, there are treatments available with one in particular at the forefront of science and sought after by every parent of a child born with SMA.
In 2019, the FDA approved the one-time infusion of Zolgensma, the first ever gene replacement therapy in the world. It has been called the "miracle cure," because, while treatments before it work to fix the SMN gene, it completely replaces it with a functioning one. It cannot undo what motor function has already been lost in Clara, but it does completely arrest any further damage from happening while being very noninvasive for someone so small. It has since taken the SMA world by storm and it has a price tag that truly shows its value at just about $2.2 million dollars, making it the single most expensive drug in the world. What makes it so valuable compared to the competition is the fact that it is a one-time intravenous prescription that takes only an hour to administer. Spinraza, our other option right now, is a series of four spinal shots into Clara's cervical spine every year for the rest of her life. If she were to miss one or if she were ever unable to make it to the hospital for the dose, her muscles would begin to deteriorate again. It is incredibly invasive and often needs surgery later on, as scoliosis may occur, to open up a cavity in her spine to continue the medication. As Clara's parents, we only want the absolute best for her, something that will give her the greatest opportunities to growth, something that will improve her quality of life the most and help her live her life to the fullest extent with this ruthless disease.
You will see that we have a short timeline to raise the funds for Clara. We, along with the whole neuromuscular team at Stanford as well as Novartis, the manufacturer for Zolgensma, have been fighting tooth and nail since we received Clara's official diagnosis last week. Although the FDA has approved the drug for use up to two years, most insurances, including our own, only cover the prescription up to 6 months for SMA type 1, despite it being a transformative treatment for all types of SMA. Clara's second birthday is this upcoming Saturday April 10th, so we are trying to do the impossible for our beautiful little girl, racing against the clock to make this happen for her.
Although she has been severely hindered by this disease, Clara is the happiest girl you will ever meet. She loves playing with her dog, Mossie, and cat, Hunter, and gives them hugs as often as they will let her. She loves doing anything outdoors, especially sitting in her swing and watching children running around at the park or in stores. She loves blowing kisses to everyone and singing songs. She loves dancing with daddy in his arms, balloons, and making every animal sound she can think of, her favorites being elephants and tigers. She only wants to be independent and loves movement and has made significant gains with her physical therapist, who has been an angel on earth for us. This past week, she has sat through countless blood draws, ultrasounds and exams and still comes out with a smile on her face ready to take on the day. She has the potential to do so much more and the hunger to keep progressing. She needs just this one missing piece with the Zolgensma and we are going to get her there, whatever it takes.
We know the odds are against us, but we have such faith that God gave her to us for a reason and a purpose, just as he gave us to her. Please help us make this hope for a cure for Clara and a better life a reality for her and donate whatever you are comfortable with. If you are religious, please keep her in your prayers. Please share this gofundme with anyone and everyone you know. Clara needs all of the help she can get. There has never been anything more worth fighting for for us as her parents and I daresay, there never will be.
Doctors notes are available on request for proof of diagnosis.
If you have any way to help, please contact us at [email redacted]
