Read the original post here. As one of the first families dosed with gene therapy, what were some of your thoughts, feelings, hopes, fears, anything you really considered as you made the decision to enroll in the trial? The idea of gene therapy was circulating at the time of our…

Written by Jessica Curran; Read the original post here. When a life alternating disease like Duchenne enters into your home it feels like your life stops while everyone else’s keeps going. I have learned that when it comes down to it, health, joy, and living a life with purpose are…

Cure Rare Disease is partnering with creators across the country to raise money for life-saving medicines for kids with rare, fatal diseases. The experience of rare disease can be overwhelming for both patients and their families. Diagnoses are often difficult, involving repeated visits to multiple specialists. Once a diagnosis is made, patients live with severe restrictions and life expectations, not to mention the tumult of having hopes raised and then dashed through…

Read the original post here. Our family was born in a beach town and the majority of our lives have played out at the shore break. Today was much of the same, another weekend at The Hook, where my nine-year-old son was out surfing. Meanwhile, my sweet seven-year-old stayed back…

Written By Justin Cohen; Read the original post here. “Do not expect much from him.” These are the words a neurologist told my parents when I was diagnosed at four-years old with a rare neuromuscular disease. The doctor did not intend them as malicious, but instead was arming my parents…

Written by Katie Kuhl; Read the original post here. My name is Katie Kuhl and I am a junior in high school right now. I am mostly focused on school and am involved in the math team, DECA, pre-med club, student council, and volunteering at my local animal shelter (pre-COVID)…

Written by Vivek Gohil; Read the original blog post here. I’m Vivek Gohil, I’m 30, and I live with Duchenne Muscular Dystrophy. My life goal is to leave a mark, make the most of life, and help others along the way. Although it’s daunting, I never worry about the future. …

Read the original blog post here. In 2020, we were thrilled to spread awareness for our cause in a variety of mediums and publications, including an appearance on the TODAY Show. We also had the opportunity to amplify the voices of rare disease parents and families through the blog we…

Meet Megan DeJarnett: a children’s book author, the CEO and founder of No Such Thing Co., and a mother of two. On January 7th, she (virtually) sat down with us at CRD and shared her journey navigating her present career and successful endeavors. Megan DeJarnett was diagnosed with spinal muscular…