What I would’ve missed out on if I’d said no to life with a disability
“Thanks for inviting me on this trip,” my 9-year-old friend Forrest said to me last weekend. His parents were in the gas station convenience store, and we were alone in the car…
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“Thanks for inviting me on this trip,” my 9-year-old friend Forrest said to me last weekend. His parents were in the gas station convenience store, and we were alone in the car…
Scholar Rock plans to have the the muscle-strengthening therapy apitegromab available commercially in the U.S. before the end of the year if the U.S. Food and Drug Administration (FDA) approves it next month…
It surprised me to open my email one night to find a message from my colleague Michael Morale. Well, it wasn’t really from Michael. It was from his Otter, a virtual assistant that had attended a company meeting…
I once read that fear is the other side of the coin to excitement, pleasure, or comfort. Without any research into whether the statement is true or false (it’s true), I…
An investigational gene therapy that targets nerve cells thats’s being developed by Exegenesis Bio was well tolerated and improved motor function in a mouse model of spinal muscular atrophy (SMA), a study shows. Based on…
In my favorite movie of all time, “The Princess Bride,” there’s a scene I love because I can relate to it and have learned a valuable life lesson from it. And today…
A man with type 3 spinal muscular atrophy (SMA) and no history of diabetes developed diabetic ketoacidosis, a severe diabetes complication, which researchers said may be linked to metabolic changes. SMA can cause major loss…
“Don’t worry, we all got the Alyssa lesson before we started working here,” the new fellow joked after having completed his second nasojejunal (NJ) feeding tube exchange on me. Though everyone in the room had a good chuckle,…
An infant with spinal muscular atrophy (SMA) type 1 and neutropenia was successfully treated with with Zolgensma (onasemnogene abeparvovec-xioi), according to a case report. Neutropenia, which refers to low levels of immune cells called neutrophils, increases…
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