dm+d

Unassigned

New Medicines

Rett syndrome in adults and children

Information

New molecular entity
Anavex
Anavex

Development and Regulatory status

Phase III Clinical Trials
None
Phase II Clinical Trials
Yes
Yes

Category

Sigma 1 receptor agonist and a muscarinic receptor modulator
Rett syndrome is an X-linked neurodevelopmental condition characterised by loss of spoken language and hand use with the development of distinctive hand stereotypies. Mutations in MECP2 are found in 95-97% of those with typical Rett syndrome. However, in atypical cases, only 50-70% of cases have mutations in MECP2. It is a moderately rare condition, but a relatively common cause of PDD. Rett syndrome affects approximately 1 in 10,000 live female births [1].
Rett syndrome in adults and children
Oral

Early (MCI and mild) Alzheimer's disease

Information

New molecular entity
Anavex
Anavex

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
None

Category

Sigma 1 receptor agonist and a muscarinic receptor modulator
Alzheimers disease is the most common form of dementia. It is estimated to affect 520,000 people in the UK. Dementia affects 7.1% of the population over the age of 65, and prevalence increases with age [1].
Early (MCI and mild) Alzheimer's disease
Oral