Movement Disorders
PRAX-944
Program Detail
Currently in development for patients with early-onset SCN2A-DEE.
PRAX-944 : Introduction
Essential Tremor (ET) is a progressive and debilitating movement disorder that significantly disrupts daily living through loss of function. ET is characterized by action tremors, which are involuntary rhythmic movements, especially in the upper limbs. The impacts on daily life and overall functioning may trigger anxiety, depression, and stress that may further exacerbate ET and deepen the impact on quality of life. There are no specific treatments developed for ET.
Praxis is developing treatments specifically designed for ET to allow people to fit the right therapy to their needs to realize improved outcomes
PRAX-944 is a novel selective T-type calcium channel blocker designed to block abnormal neuron burst firing in the CTC circuit correlated with tremor activity.
In clinical trials to date for ET, treatment with PRAX-944 up to 120 mg/day has been generally well tolerated and showed both improvements in activities of daily living and a reduction in tremor amplitude.
Mechanism of action
SCN2A variants occur when a protein coding sequence causes a strand of mRNA to undergo a missense mutation. SCN2A missense mutations are associated with severe seizure disorders.
PRAX-222 is an antisense oligonucleotide (ASO) designed to down-regulate SCN2A expression in patients with early-onset SCN2A-DEE.
Key Upcoming Milestones
Next steps for PRAX-944 Clinical Program
Medical Papers + Presentations
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