Expert Interview
Discussing the potential of uniQure's gene therapy AMT-130 and PTC Therapeutics candidate PTC518 in Huntington's Disease.
Ticker(s): QURE, PTCTInstitution: University of Louisville
- Professor of Neurology, Medical Geneticist, Endowed Chair of Parkinson’s Disease Research, & Director, Movement Disorders Program at University of Louisville.
- Manages 90 patients with Huntington's disease and 35 patients with Friedreich’s Ataxia.
- Research interests are focused on understanding the underlying genetic basis of hereditary spastic paraplegia and essential tremor; clinical interests include surgical therapies for Parkinson's disease, essential tremor and dystonia, and botulinum toxin procedures for dystonia and spasticity.
PTC recently added additional cohorts to their trial who have less severe disease. How do you think these cohorts will respond to treatment? They also added cUHDRS as an endpoint at 12 months. Is that a long enough duration to see clinical benefit?
Added By: userfeb5f22bWhich is better: targeting the striatum (uniQure) or systemic distribution across the brain (PTC)?
Added By: userfeb5f22bPTC has stated that they are aiming for 30-50% lowering of HTT in the brain. Is that a reasonable target?
Added By: userfeb5f22bAre You Interested In These Questions?
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