
Katherine Mathews
Pediatric neurologist and professor of pediatrics and neurology at the University of Iowa Carver College of Medicine, leading expert in neuromuscular disease and muscular dystrophy research.
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Sep 30, 2026
176: BBP-418 and the Changing Treatment Landscape of LGMD
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Marco MeglioHOST
So give a little bit of breakdown on what the clinical data showed for this therapy and just what we've seen from the findings thus far to support it.

Katherine MathewsGUEST
The study is actually a three-year study, which is one of the problems with doing trials in limb girdle muscular dystrophy.

Katherine MathewsGUEST
But So it's a three-year study, but they had specified when they created the study because they knew that three years is a really long time to ask people to be in a double-blind study.

Katherine MathewsGUEST
They pre-specified that they were going to analyze a subset of the data after more than half the people had been in the study for a year.

Katherine MathewsGUEST
So what they reported at the MDA meeting was the 12-month data for the subset of people.

Marco MeglioHOST
But what makes LGMD so difficult to treat? Just provide a little bit of context for our clinical community on some of the challenges that we've had with clinical trials and finding an effective agent for a patient population that doesn't have any FDA-approved therapies right now.