The experimental drug, Cabaletta, improved muscle function and reduced swallowing difficulty in patients’ suffering from oculopharyngeal muscular dystrophy.
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BioBlast said the drug improved patients’ muscle function while also reducing difficulty in swallowing.
Oculopharyngeal muscular dystrophy is a rare disease where patients develop swallowing difficulties, which could lead to death in severe cases.
The company said it would discuss results from this study and a second mid-stage study with the U.S. Food and Drug Administration before it started a late-stage study.
Source-Medindia