Drug intelligence / Profile preview

autologous myoblast graft

Development stage
Unknown
Lead developer
CHU Caen Normandie
Modality
Cell Therapies
Administration
Intramuscular
01

Overview

Autologous myoblast graft is an experimental cell therapy developed by the University Hospital, Caen, for the treatment of oculopharyngeal muscular dystrophy (OPMD). The procedure involves harvesting healthy muscle progenitor cells (myoblasts) from the patient's own unaffected muscles, such as the quadriceps or sternocleidomastoid, and expanding them in vitro. These expanded cells are then injected into the affected muscles, specifically the levator palpebrae superioris to treat ptosis or the pharyngeal muscles to treat dysphagia. The therapy aims to regenerate or reinforce the weakened muscle tissue, thereby restoring muscle function and improving clinical symptoms associated with the genetic disease.

Other names
autologous myoblast transplantationmyoblast transfer therapymyoblast autologous graft

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