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Palvella Therapeutics.
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  5. Palvella - Model QTORIN - Microcystic ...

Palvella - Model QTORIN - Microcystic LM

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QTORIN™ rapamycin 3.9% is a novel targeted topical therapy being studied for Microcystic Lymphatic Malformations (Micro LM). Palvella is enrolling a phase 2 clinical study for people with Micro LM.
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Micro LM is a rare genetic disease of the lymphatic system driven by a genetic defect in the PI3K/AKT/mTOR pathway. Microcystic LMs often have a cutaneous component and clinically present with lymphorrhea, bleeding, pain, and functional deficits. Infections of malformations can occur and may lead to cellulitis of surrounding tissues or severe, life-threatening infections. While most individuals are diagnosed early in life, the disease can become more pronounced as an individual ages and goes through puberty.

There are no FDA approved treatment for Microcystic Lymphatic Malformations.

QTORIN™ rapamycin 3.9% is designed to target the PI3K/AKT/mTOR pathway, which is overactivated in Micro LM. QTORINTM rapamycin 3.9% topical gel delivers rapamycin deep into the dermis, the area where the disease originates.