Mifepristone, a glucocorticoid blocker, increased survival and muscle size in a mouse model of spinal muscular atrophy (SMA), according to a recent study. Combining mifepristone with a gene therapy construct similar to the one used in Zolgensma and Itvisma, which is intended to increase levels of…
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Our daughter, Charlie-Anne, was born on June 22, 2009. When the doctor handed her to me, I was shocked to see a full head of beautiful, red hair. I was instantly in love. I had been diagnosed with premenstrual dysphoric disorder years before my pregnancy, so regular mental…
Every morning, after relieving the night nurse, my mom walks toward my bed. “Snookums!” she lovingly calls out when she sees that my eyes are open. It’s a nickname that comes straight from her heart. Everything my mom does for me, which is a lot, comes from that same place.
Our granddaughter, Clara, turns 10 this month. She loves choosing themes for her birthday parties, and “PopPop,” as she calls my husband, Randy, challenges himself to design and bake elaborate cakes to match. This year’s theme is based on the Broadway musical and film “Wicked.”…
Treatment with Zolgensma (onasemnogene abeparvovec-xioi) improved motor function in children with spinal muscular atrophy (SMA) who needed tracheostomy, without posing new safety risks, a real-world study showed. Tracheostomy is a surgical procedure that creates an opening in the windpipe, often necessary when a ventilator is used to assist…
Seeking inspiration for this week’s column, I asked: “Hey, Mom. What is a favorite memory you have of our family traditions during the holidays?” “That’s easy,” came the surprisingly immediate response. “Sugar cookies.” The two words brought a smile and a flood of nostalgia to me. Every December, for as…
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