
Loss of function gene mutations in SCN2A: Implications and opportunities for therapeutic intervention
Professor Steven Petrou explains how mutations in the SCN2A gene can lead to loss of function, and how treatments could increase function.
Professor Steven Petrou explains how mutations in the SCN2A gene can lead to loss of function, and how treatments could increase function.
Antisense oligonucleotides (ASOs) are being developed as treatments for rare genetic disorders such as SCN2A. What are they and how do they work?
Prof Steven Petrou and Dr Kiran Reddy discuss the RC-222 program developing an ASO treatment for gain-of-function SCN2A epilepsies.
Speakers: Prof. Steven Petrou, Director of the Florey and RogCon, Chief Scientific Officer, and Dr. Kiran Reddy, CEO of Praxis Precision Medicines. At the 2018
We talk with Dr Kiran Reddy about the plans for the RC-222 treatment program with the goal of developing a treatment for SCN2A gain-of-function epilepsies.
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