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Katherine Mathews

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.

Sep 30, 2026

3:50
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.
3:58
The interim analysis results were reported at the MDA meeting in March.
4:05
So that's what I will be referring to.
4:10
The study is actually a three-year study, which is one of the problems with doing trials in limb girdle muscular dystrophy.
4:18
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.
4:32
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.
4:43
So what they reported at the MDA meeting was the 12-month data for the subset of people.
7:35
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.

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