The days are long, but the years are short. I’ve often heard that sentiment from parents whose children seem to grow up too fast. I’ve had several moments myself over the years of parenting my seven children. I had one of those moments yesterday when my oldest son, Max, who…
A form of congenital muscular dystrophy caused by mutations in a gene called GGPS1 may not always lead to hearing loss, contrary to what has been described previously, according to a recent study. The report, “GGPS1-associated muscular dystrophy with and without hearing loss,” was published as a brief…
Note: This column includes thoughts about suicide. Resources for help are listed at the end of the column. I delivered my first research presentation at the College of Alice & Peter Tan (CAPT) at the National University of Singapore (NUS) on Sept. 17. In March, my favorite lecturer from my…
Sarepta Therapeutics has submitted an application to the U.S. Food and Drug Administration (FDA) asking the agency to grant accelerated approval to the gene therapy SRP-9001 (delandistrogene moxeparvovec) as a treatment for Duchenne muscular dystrophy (DMD) in patients who are able to walk. “Every hour of every…
Duchenne muscular dystrophy (DMD) is a progressive disease, but for me, the mother of three sons with DMD, things have been more nuanced. The grief I experience from being associated with a rare, chronic disease has been anticipatory, erratic, and constantly cyclical. It did not start small and grow…
Dystrogen Therapeutics’ experimental cell therapy, DT-DEC01, continues to show signs of efficacy in three boys with Duchenne muscular dystrophy (DMD), who exhibited improved muscle strength and motor function after six months in a clinical trial. All patients showed signs of clinical improvements irrespective of their mutation in the…
A first patient has been dosed in Denmark as part of a Phase 1/2 trial of ATA-100, Atamyo Therapeutics’ one-time gene therapy for a specific form of limb-girdle muscular dystrophy called LGMD-2I or LGMD-R9. “This is an exciting milestone for our company but most importantly, if this clinical trial is…
As a mom to three sons with Duchenne muscular dystrophy, Betty Vertin sometimes feels that she needs to meet their needs at the expense of her own. Recognizing when she needs to take some time away for self-care is vital to her perseverance as a caregiver and her mental health as a mom, she says.
The beginning of a new college football season also means it’s time for Coach to Cure MD (CTCMD) — a campaign aimed at calling attention to Duchenne muscular dystrophy (DMD) and raising research funds for a cure. Slated for Sept. 24, the 15th annual event is a…
Last week, I started a new job as a part-time community partnerships executive with K9Assistance, Singapore’s first assistance dog advocacy organization. Assistance dogs are service dogs trained to help disabled people perform tasks to mitigate their impairments. As a Duchenne muscular dystrophy survivor, I’ve always been intrigued…
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