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1 month ago | 
musculardystrophynews.com | Marisa Wexler |Betty Vertin
I recently had the opportunity to attend a Singaporean theater production titled “Supervision,” thanks to the SingHealth Patient Advocacy Network (SPAN). SPAN is a collective of patients and caregivers who provide feedback to improve healthcare here in Singapore. I’ve been part of it since 2022. The invitation to the play was a thank-you for volunteering with several healthcare projects, including reviewing fact sheets on chronic illnesses for the Health Ministry’s Agency for Care Effectiveness.
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1 month ago | 
musculardystrophynews.com | Marisa Wexler |Betty Vertin |Margarida Maia
KER-065, a potential treatment for Duchenne muscular dystrophy (DMD) and other neuromuscular diseases, showed a good safety profile in a Phase 1 clinical trial. No serious side effects were seen in the trial, which tested single and multiple doses of KER-065 in healthy volunteers, and no major safety issues were noted. Data also indicate that KER-065 is working as designed, according to developer Keros Therapeutics.
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1 month ago | 
musculardystrophynews.com | Betty Vertin |Lindsey Shapiro |Andrea Lobo
“Mommy, hold me. Mommy, hold me.” These were the words I heard repeatedly from my 3-year-old daughter, Callie, during the four days we spent in the neuromuscular clinic with her older brothers last week. It wasn’t normal for her to want to be held. I’ve never traveled with a stroller for her because she prefers to ride on the footrests of her brothers’ power wheelchairs.
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1 month ago | 
musculardystrophynews.com | Marisa Wexler |Betty Vertin |Andrea Lobo |Steve Bryson
RGX-202, a one-time gene therapy designed to treat Duchenne muscular dystrophy (DMD), has been well tolerated in an ongoing clinical trial, with no serious side effects reported. Interim data from the Phase 1/2 part of the trial, which is sponsored by RGX-202’s developer Regenxbio, also indicate that RGX-202 appears to be working as intended, robustly increasing the production of microdystrophin and leading to improvements in motor function for DMD boys.
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2 months ago | 
musculardystrophynews.com | Betty Vertin |Marisa Wexler
Superheroes were a large part of my life less than a decade ago. I am a boy mom. We had four sons in five years, and our house quickly became a lived-in museum of all things boys. Power Rangers, X-Men, and Marvel and DC Comics characters were like family members for a few years. Those superheroes joined our family of nine. I share seven children with my husband, Jason: Lexi, 23, Max, 19, Chance, 17, Rowen, 16, Charlie, 14, Mary, 10, and Callie, 3.
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2 months ago | 
musculardystrophynews.com | Robin Stemple |Marisa Wexler |Betty Vertin
I’ve played music since I was 5 years old. My first professional playing job was as an accordionist in a Polish polka band when I was 13. My accordion at the time was a full-size Excelsior 120 bass model that weighed about 35 pounds. My professional playing career took a lot of turns over the next 18 years. I enjoyed playing in a rock group, a polka band, a disco group, a country band, and, eventually, a prototypical “wedding” band that did a little bit of everything.
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2 months ago | 
musculardystrophynews.com | Andrea Lobo |Marisa Wexler |Betty Vertin |Hawken Miller
Adolescents and young adults with Duchenne (DMD) or Becker muscular dystrophy (BMD) who have more siblings typically report being better prepared to transition to adult life, a new study has found. Findings show people with DMD or BMD had the most difficulty with activities of daily living, education, and employment when navigating adulthood, and fewer needed help with healthcare, housing and transportation, and independent life and autonomy.
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Feb 4, 2025 | 
musculardystrophynews.com | Margarida Maia |Betty Vertin |Andrea Lobo |Lindsey Shapiro
Entrada Therapeutics announced it has been cleared in the U.K. to start a Phase 1/2 clinical trial of ENTR-601-44, at increasing doses, in Duchenne muscular dystrophy (DMD) patients with a mutation in the DMD gene amenable to exon 44 skipping. With this decision by U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA), which follows positive findings from a study in healthy adults, Entrada plans to initiate the trial later this year.
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Jan 31, 2025 | 
musculardystrophynews.com | Betty Vertin |Steve Bryson
I read a quote the other day on Facebook: “When you are raising babies, you feel how long the days are. When raising teens, you feel with every single bone in your body how short the years are.”Days later, I still carry those words with me as a mom of seven children: Lexi, 23; Max, 19; Chance, 17; Rowen, 16; Charlie, 15; Mary, 10; and Callie, 3. There are 20 years between my oldest and youngest children. I think the quote is an apt description of my experience with them.
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Jan 24, 2025 | 
musculardystrophynews.com | Betty Vertin |Marisa Wexler
Parenting is the most challenging job I’ve ever had. When my role as a parent expanded into caregiving for three of my children, challenging didn’t even begin to describe the path that lay in front of us. I share seven children with my husband, Jason: Lexi, 23, Max, 19, Chance, 17, Rowen, 16, Charlie, 14, Mary, 10, and Callie, 3. Max, Rowen, and Charlie are all living with Duchenne muscular dystrophy (DMD).
