CORDIS Project
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MyoPax is developing targeted regenerative therapies for muscular dystrophies using its Satgeno platform. By harnessing stem cell technology, the project aims to deliver gene repair therapies that restore muscle function and improve the quality of life for patients suffering from these debilitating disorders.
Muscular dystrophies (MDs) are a group of about 50 different monogenic disorders characterized by progressive, irreversible muscle wasting, leading to loss of autonomy and premature death.
Patients rely on palliative care; the disease burden is immense.
Current gene therapy approaches do not address the underlying genetic causes.
The challenges to develop gene therapies for MDs include the genetic variability and the lack of a safe drug delivery methods to muscle.MyoPax is a clinic-ready company…
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