Taysha IPO Presentation Deck
Residual Hex A activity determines the
severity of GM2
Small increases in Hex A activity may
lead to significant improvements in
clinical outcomes and quality of life
Infantile onset is the most severe
form of GM2
- Infantile forms may die within the
first 4 years of life, and juvenile onset
patients rarely survive beyond mid-
teens
Infantile (onset < 6 months)
- Startle response
Hypotonia
Cherry-red macula
Seizures
. Macrocephaly
- Progressive visual loss
- Death 3-5 years
<0.1%
F.
Juvenile (onset 1.5-5 years)
- Cerebellar ataxia
Decreased cognition
-Progressive weakness
Visual loss
- Seizures
- Death in mid-teens
Early adulthood onset
- Progressive weakness
Cerebellar ataxia
-
*
Normal life span
0.50%
2-4%
Residual Hex A Enzyme Activity
Psychosis
Decreased cognition
Age at death variable, normal
life span possible
5-10%
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