dm+d

Unassigned

New Medicines

Confirmed infantile neuroaxonal dystrophy (INAD), homozygous for PLA2G6 deficiency in children aged 18 months to 10 years

Information

New molecular entity
Retrotope
Retrotope

Development and Regulatory status

None
None
Phase III Clinical Trials
Yes
Yes

Category

INAD is an inherited condition that leads to neuronal cell death due to failure to clear toxic products from lipid peroxidation (LPO). RT001 is a synthetic linoleic acid (LA) designed to combat the oxidative stress & cellular degeneration arising from LPO
INAD is an ultra-rare, progressive, fatal, infant genetic neurological disorder caused by an inborn genetic defect caused by-products of lipid peroxidation (LPO), creating a “death signal” resulting in rapid neuronal cell death in the infant’s brain. This causes irreversible loss of vital functions and progresses to death. Prevalence is unknown, but more than 150 cases have been described (globally), of which the majority are classic INAD. [2]
Confirmed infantile neuroaxonal dystrophy (INAD), homozygous for PLA2G6 deficiency in children aged 18 months to 10 years
Oral