Late afternoon sunlight drifts into the house, brightening the blue paint on the walls. The room feels cheery and welcoming. Wicker baskets hold soft blankets, pillows cushion the couch, and dogs are sleeping on the rugs. I love walking into this room, and every room, in my home, which I…
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne muscular dystrophy (DMD) that’s being developed by Precision Biosciences. The designation provides companies with extra incentives to develop experimental therapies to treat rare diseases that mainly affect…
I’m an early riser. As I mentioned in a past column, I like to wake up early, get dressed, make coffee, and start the day with some quiet prayer and devotion time. However, I’m also a very sound sleeper, with some help from melatonin, sleep medication, and a comfy…
The U.S. Food and Drug Administration (FDA) is due to convene an advisory committee meeting next month to review the data on deramiocel, an experimental cell therapy up for approval to treat heart disease in people with Duchenne muscular dystrophy (DMD). The FDA has informed Capricor Therapeutics…
Here in the Midwest, June brings warmer temperatures. I live in Nebraska, where warmer weather is welcome after bitterly cold winters and brisk, windy springs. It’s also necessary for the crops growing in the large fields that line most of the highways. I wait all spring for the temperature to…
Living with Duchenne muscular dystrophy (DMD) has taught me countless lessons, especially the power of connection, creativity, and celebrating every form of love. Last Saturday, I attended the 11th annual concert of The Purple Symphony with my girlfriend, Amanda, and my new caregiver, Julie. Titled “Perhaps, It’s Love,”…
A second person with Duchenne muscular dystrophy (DMD) has died of acute liver failure after being treated with the one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl). A first death was announced in March and both occurred in people with DMD who were no longer able to walk. Following…
I’m a mother of seven and a caregiver to the three of my sons who have Duchenne muscular dystrophy (DMD). My oldest daughter, Lexi, 24, lives a few hours away with her husband. I live in rural Nebraska with my husband, Jason, and our six youngest children: Max, 19;…
The European Comission has conditionally approved Duvyzat (givinostat) to be taken alongside corticosteroids for treating people who are 6 and older with Duchenne muscular dystrophy (DMD). “This milestone means that a broad range of patients with DMD have access to a new treatment,” Francesco De Santis, president of…
I’m between two anniversaries. Today is the 36th anniversary of the collision I had with a drunk driver that cost me my eyesight and almost cost me my life. Saturday will be my 45th wedding anniversary. The car crash happened on the evening of June 11, 1989. I was returning…
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