and her mother Mayra
We are focused on developing treatments for diseases that impact the MC4R pathway. These include:
POMC/PCSK1/LEPR deficiencies
Rare biallelic variants of the POMC, PCSK1, and LEPR genes can result in deficiencies of certain proteins and enzymes that help the body regulate food intake, resulting in insatiable hunger (hyperphagia) and early-onset obesity.
Bardet-Biedl syndrome (BBS)
BBS is a syndromic ciliopathy, and a rare, potentially underdiagnosed genetic disease that leads to a variety of clinical characteristics, including insatiable hunger (hyperphagia) and early-onset obesity, causing profound challenges for patients and their caregivers.
Acquired hypothalamic obesity (HO)
A rare disease caused by injury to the hypothalamus that disturbs the MC4R pathway, typically brought on by certain brain tumors and treatment of them, resulting in insatiable hunger (hyperphagia) and rapid‐onset, severe obesity.
Additional rare MC4R pathway diseases
Rare, highly impactful variants in just one of several different genes associated with the MC4R pathway can result in loss of function in that pathway, potentially leading to insatiable hunger (hyperphagia) and early-onset, severe obesity.
Additional neuroendocrine focus area
Congenital hyperinsulinism (CHI)
A rare condition that causes the body to produce too much insulin, resulting in persistent hypoglycemia (low blood sugar) that can cause seizures, coma, neurological damage, and even death.
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