Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A
NULIBRY is a first-in-class synthetic cPMP substrate replacement therapy to reduce the risk of mortality in patients with MoCD Type A
MoCD Type A is a rapidly progressive autosomal recessive inborn error of metabolism resulting in toxic sulfite levels causing neurologic sequelae
NULIBRY is a first-in-class cPMP substrate replacement therapy to reduce the risk of mortality in patients with MoCD Type A.
The company is also looking at an inorganic opportunity over the next two years to have a commercially ready footprint in the US
Nulibry is approved by the U.S. Food and Drug Administration (USFDA) to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening paediatric genetic disorder
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