BioBlast Skips To Phase III In Rare Form Of Muscular Dystrophy
This article was originally published in The Pink Sheet Daily
Biotech plans Phase III program for I.V. sugar formulation Cabaletta in oculopharyngeal muscular dystrophy, after reporting limited, proof-of-concept data for the rare, debilitating swallowing disorder.
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Patients with the most severe type of disease were all randomized to treatment with the study drug, which may have had a negative impact on outcome, the Israeli company suggested.