Michael Snape, PhD, Chief Scientific Officer at AMO Pharma describes the history of his company and its current pipeline that is largely focused on rare neurodevelopmental conditions, including congenital myotonic dystrophy, Phelan-McDermid syndrome, and Rett syndrome.
Congenital myotonic dystrophy is a rare inherited disease in which babies have weak muscles and weak muscle tone, as well as severe cognitive disabilities. AMO Pharma is developing a glycogen synthase kinase 3 beta (GSK3ß) inhibitor to treat this condition.
Phelan-McDermid syndrome is also a rare inherited disease. Common are intellectual and behavioural patterns that can mimic those seen in autism. The disease is due to mutations in chromosome 22. AMO Pharma is developing a RAS-ERK inhibitor to treat this condition.
Rett syndrome is a progressive, neuro-developmental condition that primarily affects girls. Most appear normal in early development but eventually start to show rapid regression in language and motor skills. AMO Pharma is developing a glutamate modulator to treat this condition.
To learn more about these and other neurological conditions, click here.