mTORC1 Inhibitors for Certain Rare Diseases

Lymphangioleiomyomatosis (LAM) and tuberous sclerosis are rare diseases caused by specific genetic defects in the mTORC1 activation pathway, resulting in an uncontrolled hyperactivity of this complex that leads to abnormal cell and tumor growth. Navitor’s therapeutics can restore more normal function by specifically inhibiting the central process leading to these diseases.

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