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.
About Micro LM:
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.
Targeting the underlying cause of Micro LM
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.
Interested in Enrolling in our Clinical Study?
Palvella is actively enrolling a clinical study for Microcystic Lymphatic Malformations! Please use the link below to learn more about participating in this study.
Clinically confirmed diagnosis of Microcystic Lymphatic Malformation
Age 13 years or older
If less <18 years of age, the participant and legal guardian must provide written informed consent/assent prior to any study procedures, including providing a separate consent for obtaining photographs.
The microcystic LM must have a defined area of >~25 cm2, with no more than three total areas to treat. The total area cannot be > ~200 cm2
Able and willing to comply with all protocol-requiring activities
Resources for Microcystic Lymphatic Malformations
Vascular Birthmarks Foundation
The Vascular Birthmark’s Foundation is the leading patient advocacy group for people with all types of vascular birthmarks, including lymphatic malformations, connecting patients, researchers, and physicians throughout the world in a united effort to help those with Vascular Birthmarks.
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