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. Author manuscript; available in PMC: 2011 Nov 30.
Published in final edited form as: Trends Mol Med. 2009 Feb 21;15(3):89–100. doi: 10.1016/j.molmed.2009.01.001

Table 1.

Neurodegenerative disorders and US FDA-approved drugs

Disorder Affected neurons Age of
onset
Sporadic
or genetic
Familial
disease
genes
Drug Mechanism of action or target Date
of US
FDA
approval
Effect
AD Cortical and hippocampal neurons >65 95%
sporadic,
5% familial
APP

PSEN1

PSEN2
Memantine
(Namenda)
Donepezil
(Aricept),
Galantamine
(Razadyne),
Rivastigmine
(Exelon).
Blocks NMDA receptors, reduces excitotoxicity
Acetylcholinesterase inhibitors.
Increase concentration of Ach in the brain
2003

1996

2001

2000
Mild improvement in cognitive measures
Mild improvement in cognitive measures
PD Dopaminergic neurons in substantia nigra pars compacta >65 95%
sporadic,
5% familial
Synuclein
LRRK2
Parkin
PINK1
DJ-1
L-Dopa
(Levodopa)
Increases amount of dopamine in substantia nigra neurons 1970 Symptomatic benefit
ALS Motor neurons 40–60 95%
sporadic,
5% familial
SOD1 Riluzole
(Rilutek)
Antiglutamate (activator of glutamate uptake, inhibitor of NMDAR and Na+ channels) 1995 Extends survival by several months
HD Striatal medium spiny neurons 40–50 100%
familial
Huntingtin Tetrabenazine
(Xenazine)
Antidopamine (VMAT2 inhibitor, reduces amount of released dopamine) 2008 Reduction in chorea
SCAs Various brain regions involved in motor control 40–50 100%
familial
Ataxins N/A N/A N/A N/A