Sarepta Therapeutics has executed an exclusive license agreement for a gene therapy candidate that aims to treat limb-girdle muscular dystrophy type 2A (LGMD2A), the company announced. The investigational therapy, called calpain 3 or CAPN-3, was developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital, in Columbus,…
This week, I wanted to visit the reason I started writing this column. However, to describe that rationale, I need to revisit the moments soon after I received my diagnosis. I was diagnosed with limb-girdle muscular dystrophy (LGMD) in the late 2000s. I remember my primary physician calling…
The U.S. Food and Drug Administration (FDA) has cleared Avidity Biosciences to start a Phase 1/2 clinical trial testing the company’s investigational therapy AOC 1001 in adults with myotonic dystrophy type 1 (DM1). “The FDA clearance to proceed with our Phase 1/2 clinical trial for AOC 1001…
My 2-year-old son, Alfie, lives with LAMA2-related muscular dystrophy. Obviously, it is hard, stressful, and heartbreaking to raise a child with a disability. I wouldn’t wish this condition upon anyone. From his premature birth to his diagnosis, the experience has been a huge learning curve for us, to say…
CureDuchenne has teamed up with Brigham and Women’s Hospital, in Boston, to launch the nation’s first free newborn screening initiative for Duchenne muscular dystrophy (DMD). The partnership establishes Brigham and Women’s as the only U.S. birth hospital to offer parents the choice of screening their new baby for…
After being diagnosed as a carrier of x-linked myotubular myopathy (XLMTM) and losing her infant twin boys to the disease, Ashley Walker was driven into an episode of alcoholism. What she didn’t know then was that her liver was affected severely by the disease, which eventually resulted in an…
A treatment combination of two blood pressure medicines can help slow heart function decline in males with Duchenne muscular dystrophy (DMD), a single center study suggests. These findings support international guidelines that recommend the use of cardiac medications to help stabilize heart function in DMD. The study, “…
As I noted in previous columns, everyone is different. You are who the other is not. This philosophy also applies to the life experiences that lead to a diagnosis. As a child, I was often referred to as clumsy. While I vehemently denied it in my mind, it…
A Phase 1b clinical trial evaluating Astellas Pharma’s ASP0367 is recruiting boys with Duchenne muscular dystrophy (DMD), ages 8–16, at six U.S. sites. The ongoing trial (NCT04184882) is evaluating the safety and tolerability of the investigational oral therapy, which is designed to improve muscle function in patients with DMD,…
Last week, I traveled to the headquarters of my employer, Bionews, the publisher of Muscular Dystrophy News Today. The trip took me from Newport Beach, in Southern California, to Pensacola, in the Florida Panhandle. While it was an interesting experience, it wasn’t without its challenges, especially with Duchenne…
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