BioBlast Pharma Ltd. of New Haven, Connecticut, is committed to developing treatments for patients living with rare genetic diseases.
Current research programs have scientists working on studies of Cabaletta, a medication for the treatment of oculopharyngeal muscular dystrophy (OPMD).
OPMD is marked by drooping of eyelids, weakness of the muscles that control eye movement, difficulty swallowing and weakness and deterioration of the tongue. As the disease progresses, the patient develops weakness in the limbs.
"Our primary objective is to develop a robust clinical program and regulatory dossier for Cabaletta," Colin Foster, BioBlast's president and CEO, said. "With the accumulating knowledge from our open-label, interim HOPEMD Phase 2 clinical study, we believe that a double-blind, placebo-controlled Phase 2b clinical study will enhance our clinical development program for Cabaletta."
BioBlast scientists expect to update the research and development plans for Cabaletta in early 2016.
More information about BioBlast and its portfolio of products is available at www.bioblast-pharma.com.
