My wife, Wendy, and I have always loved having folks over to the house, especially during the holidays. We used to enjoy visiting our friends, as well. Having friends over was short-circuited when we moved to Pittsburgh in January, leaving a big part of our circle back in Shanksville,…
The HOPE-3 clinical trial testing Capricor Therapeutics’ experimental cell therapy CAP-1002 for Duchenne muscular dystrophy (DMD) can continue as planned, the company announced. That’s the result of an interim futility analysis, which evaluates whether it is mathematically possible for a trial to achieve its goal — if not, the trial…
I’m a small-town girl. I love that I can get from one end of my hometown in rural Nebraska to the other in a few short minutes, that there is never traffic, and that the only time I get stuck in a crowd is if I go to the county…
Corticosteroids may cause fewer unwanted brain changes in boys with Duchenne muscular dystrophy (DMD) if they are given on and off at 10-day intervals, rather than at daily doses, an imaging study found. “Brain volumetrics [volume measurements] are more profoundly affected in patients treated with a daily corticosteroid…
As this column is published, it’s the eve of the fourth anniversary of my brother Isaac‘s death. He passed away on Dec. 7, 2019, from heart disease related to his Duchenne muscular dystrophy (DMD), which I have, too. I feel it’s significant, even rare, that someone with…
The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to Edgewise Therapeutics‘ EDG-5506 as a potential treatment for Duchenne muscular dystrophy (DMD). EDG-5506 also was awarded FDA orphan drug status for Becker muscular dystrophy (BMD) as a possible…
Note: This column describes the author’s sons’ experiences with a trial dose of Translarna (ataluren) in the U.S. Not everyone will have the same response to treatment. Consult your doctor before starting or stopping a therapy. I’m a mother to three sons with Duchenne muscular dystrophy (DMD): Max, 18,…
Growth hormone (GH) or testosterone can better protect against spinal fractures than a common osteoporosis medication alone in boys with Duchenne muscular dystrophy (DMD) or severe Becker muscular dystrophy (BMD), according to a long-term study. Used to prevent or lessen osteoporosis, or weak and brittle bones, zoledronic acid (ZA) is…
I was diagnosed with facioscapulohumeral muscular dystrophy (FSHD) at age 14, when I was a freshman in high school. But even before that, I had begun adapting to my physical challenges. My FSHD journey actually started with a counselor from the Pennsylvania Office of Vocational Rehabilitation who visited…
NS Pharma has opened a program for people with Duchenne muscular dystrophy (DMD), and their caregivers and family members, to share insights that might help others living with this most common form of muscular dystrophy. Called Duchenne Heroes, it relates the experiences of patients and…
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