Last Saturday, I attended a focus group on behalf of a local patient advocacy community of which I’m a member. The agenda for discussion was a nationwide allied health strategy here in Singapore to be implemented in coming years. For those unfamiliar with the term allied health, Kent State…
AMO Pharma has announced it will conduct a Phase 3 trial of AMO-02 (tideglusib), its investigational oral therapy for adult-onset myotonic dystrophy type 1 (DM1). The decision follows a recent meeting with the U.S. Food and Drug Administration (FDA) to review data from the Phase 2/3…
Spring is by far the busiest season for my family. I have seven children who range in age from 2 to 23 years old, and three of them, Max, 18, Rowen, 15, and Charlie, 13, are living with Duchenne muscular dystrophy (DMD). Caregiving for my three sons with…
A $250,000 award from the Parent Project Muscular Dystrophy (PPMD) will support the establishment of Baby Duchenne, a collaborative clinical research network for babies with Duchenne muscular dystrophy (DMD) diagnosed via newborn screening (NBS) programs in New York state. Under the direction of Bo Hoon Lee, MD, from…
Researchers have discovered a new, unrecognized type of muscular dystrophy that’s caused by inherited mutations in the SNUPN gene, a study reports. Most people who carry the mutations develop symptoms of muscle weakness before age 2, and the muscles of the upper arms and legs are mainly affected. “This…
I have three sons with Duchenne muscular dystrophy (DMD). Max, 18, Rowen, 15, and Charlie, 13, are part of a bustling family, joined by four other siblings: Lexi, 22, Chance, 16, Mary, 9, and Callie, 2. We have no sets of twins or multiples, yet I have often grouped…
Most people with myotonic dystrophy type 1 (DM1) develop heart problems, according to a Danish study that followed patients for about 10 years. “Life-long cardiac screening is crucial, and we recommended that repeated follow-up is performed,” its researchers wrote in “Natural history of cardiac involvement in myotonic dystrophy type 1 – Emphasis…
In my last column, I wrote about the increasing difficulties I’m having safely navigating my home using the “furniture surfing” technique. I wondered whether it was time that I break out the walker and give it another try. I also mentioned my plans to contact my doctor to discuss…
Glucocorticoid use can help maintain breathing ability and arm function in adults with Duchenne muscular dystrophy (DMD), a new study reports. Importantly, the study found that glucocorticoid treatment was shown to be helpful even when only considering the time after patients had lost the ability to walk — which…
It’s now tornado season here in Nebraska, which is part of the U.S. Tornado Alley. I could write a column about Duchenne muscular dystrophy (DMD) being like a tornado in the way it came tearing through the life we’d established when my three sons — Max, 18,…
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