“Vroom, vroom, vroom” trails my son in the hallway at school. “Whoa, there. Do you guys have a license for those things?” an older gentleman says as my family navigates our way through a shopping area. “You drive that well,” a stranger remarks. Those are just some of the comments…
The European Medicines Agency (EMA) has started reviewing an application to market givinostat for Duchenne muscular dystrophy (DMD). “Now that our market authorization application is under review, we are looking forward to working with the regulatory authorities to bring this treatment option to patients,” Paolo Bettica, MD, PhD,…
Pamrevlumab, an antibody in FibroGen’s pipeline, did not improve motor skills in boys with Duchenne muscular dystrophy (DMD), ages 6 to 11, who could walk, according to top-line data from a Phase 3 study. The LELANTOS-2 (NCT04632940) trial tested against a placebo pamrevlumab’s safety and how…
Duchenne muscular dystrophy (DMD) can be all-consuming. It’s so big that it often takes up all the leftover space in a room. However, we’ve had years where life is bigger than Duchenne. I’m grateful to be experiencing one of them now. My life often revolves around DMD because…
September will be a busy month for patients, supporters, doctors, and researchers in the muscular dystrophy (MD) community as they recognize National Muscular Dystrophy Awareness Month. Raising awareness and critical funds for the more than 300,000 families across the country with MD and related neuromuscular diseases is the goal…
Those who read my columns regularly will know that my last one was written just after my wife, Wendy, and my daughter, Jill, took off for a vacation in Croatia, Greece, and Slovenia. Over their two-week trip, we communicated by FaceTime a lot, and they seemed to be having…
Blocking the activation of a channel in mitochondria, the cells’ powerhouses, may be a promising way to stop muscular dystrophy (MD) progression in a mouse model of the disease. “We have isolated the primary disease-causing component of muscular dystrophy to the mitochondrial permeability pore,” Jeffery Molkentin, PhD, co-executive director…
The first time the words “Duchenne muscular dystrophy” (DMD) were spoken to me, they might as well have been in a whisper. I could barely comprehend the specialist sitting across from me who was saying something was wrong with Max, my beautiful, oldest son. I remember asking questions, although…
More than 420 “Fill the Boot” events will raise money throughout Labor Day weekend to advance research and care for muscular dystrophy. The campaign results from a partnership between the Muscular Dystrophy Association (MDA) and the International Association of Fire Fighters (IAFF). The firefighters go to…
Aug. 14 isn’t just another day for my family. This year, our conversations were imbued with heartfelt remembrance as we gathered around the dinner table. We celebrated the birthday of my brother, Isaac, even though he was no longer with us physically. Isaac passed away due to heart…
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