About Molybdenum Cofactor Deficiency (MoCD) Type AMoCD Type A is a severe, ultra-rare and genetic metabolic disease affecting newborns in which a genetic deficiency of cPMP results in the inability of the body to form an essential cofactor called molybdenum cofactor, resulting in its absence. This cofactor is essential for the appropriate functioning of several critical metabolic enzymes. A deficiency or absence of this cofactor leads to accumulation of toxic molecules, including the neurotoxin sulfite. Clinically, the absence of this cofactor and the resulting build-up of sulfite in the brain lead to damage and destruction of nerve cells, brain swelling, uncontrollable seizures, catastrophic and irreversible brain damage, and ultimately, death. 2 There are currently no treatment options for patients with MoCD Type A.
FDA Grants Breakthrough Therapy Designation To CPMP Replacement Therapy For Patients With Molybdenum Cofactor Deficiency (MoCD) Type A
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