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The Rare Disease Rollercoaster: ER prepared while on an intensive trip.

Riding the Rare Disease rollercoaster comes with many unexpected twists and turns, sometimes we find ourselves in situations we never imagined. Such was the case for Blythe and her daughter Maggie, whose journey led them to an ER out of state. Blythe, a dedicated caregiver and advocate for Maggie, shares their remarkable experience with vulnerability and grace.


“With Maggie, I had a normal pregnancy and birth, but she failed her newborn hearing screening. After multiple failed attempts we were referred to audiology.  Looking back on it now, we were actually fortunate because it jump started the diagnosis process.  I also had a gut feeling in the very beginning that there was more going on.  She is our one and only child so it was all new, but I still just knew.  She didn't take a bottle easily, she struggled gaining weight, & I felt like we were missing milestones early on.  It's so hard when you feel like there could be something seriously wrong & you're ready to accept it, when everyone else around you is telling you that it's normal and is in denial."




 

When Maggie was 4 months old she had an MRI of her ears to make sure everything was structurally normal. In turn they found out that while her ears looked great, her cerebellum was very underdeveloped. 

 

At 8 months, they found out that the cause of her symptoms was a mutation in her CASK gene. While her diagnosis was hard to hear, they finally felt validated in their worries and it ended up leading them to an incredible group of families going through the same thing.

 

CASK-related intellectual disability is a disorder of brain development that has two main forms: microcephaly with pontine and cerebellar hypoplasia (MICPCH), and X-linked intellectual disability (XL-ID) with or without nystagmus. Within each of these forms, males typically have more severe signs and symptoms than do females; the more severe MICPCH mostly affects females, likely because only a small number of males survive to birth.

 

For more information about Cask related disorders: https://www.projectcask.org


"Being a caregiver and advocate for Maggie has been one of the hardest and most rewarding things I’ve ever done," Blythe reflects, her words echoing the sentiments of countless parents navigating the complex terrain of rare diagnoses. But Maggie has taught me so much about finding joy in the little bits of life and cherishing every moment. She has really put into perspective the things in life that truly matter." Says Blythe.





Their unexpected journey unfolded during a trip for an intensive, a time meant for focused care and support. However, as life often does, it threw a curveball. Maggie began feeling unwell, prompting Blythe to take swift action and seek medical attention at a local hospital. Stepping into an unfamiliar medical facility, Blythe felt a surge of apprehension. The doctors and nurses knew nothing about Maggie's diagnosis, and the absence of her medical records in their system compounded the uncertainty.


"It’s scary to be at a facility that knows nothing about your child’s diagnosis and doesn’t have her records," Blythe shares, her voice tinged with the raw honesty of a parent navigating uncharted territory.


"Thankfully, I have mejo on my phone, which came in handy to bring the medical team up to speed while I focused on Maggie."



In those moments of vulnerability, as they stood on the precipice of uncertainty, Blythe's preparedness became their lifeline. With mejo—a comprehensive medical management app—accessible on her phone, and a meticulously organized printout, Blythe was armed with the tools she needed to advocate for her daughter. Amidst the flurry of assessments and treatments, she could focus her attention where it mattered most: on Maggie, her precious daughter whose well-being was her main concern.


Blythe's words resonate with the raw emotions of countless parents who have found themselves in similar situations—struggling to bridge the gap between their child's complex medical history and the unfamiliar faces of a medical team in an ER far from home. Yet, in the face of uncertainty, Blythe's proactive approach shines as a beacon of empowerment, a testament to the resilience and resourcefulness inherent in the journey of caregiving.


As Blythe and Maggie ride all of the the complexities of Maggie's CASK disorder and the myriad challenges it presents, they do so with unwavering determination and an unbreakable bond. Theirs is a story of courage, love, and the remarkable capacity of the human spirit to rise above adversity. And in sharing their journey, they offer hope and inspiration to others walking similar paths, reminding them that, even in the darkest moments, they are never alone.


“My favorite thing about mejo is that when I'm too overwhelmed to remember ALL of the details, it's there to help me out.  You start to learn the questions that you are repeatedly asked, so I make sure to have extra notes for most of those scenarios. “ Shares Blythe


mejo is a caregiver’s assistant that provides support for caregivers, rapid insights for sponsors and deeper engagement for communities.


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