The study looks at people with Limb Girdle Muscular Dystrophy type 2I, also called LGMD2I/R9. This is a rare condition that slowly weakens the muscles around the hips and shoulders. The treatment being tested is an oral medication made of granules that contain Ribitol, referred to in the study as BBP-418.
The purpose of the study is to assess the long‑term safety and clinical benefit of the medication. Participants will take the granules by mouth each day for an extended period and will attend regular visits where doctors will check how they are feeling and perform simple tests.
During the visits, several simple assessments are done: a muscle‑function questionnaire called NSAD, a walking speed test over ten meters known as the 10MWT, a breathing test that measures how much air can be exhaled called FVC, an upper‑limb function test named PUL 2.0, a walking distance test over one hundred meters called 100MTT, and a blood test that measures a protein called CK which indicates muscle damage. These checks help researchers understand how the medication affects muscle strength, movement, and overall health over time.
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