Tag: Spinocerebellar ataxia

In a significant development for the rare disease community, the FDA approved the first-ever treatment for Friedreich's ataxia in early 2023, offering a major breakthrough after years with no disease-specific drugs. This milestone, along with other promising therapies currently under review, provides a clearer answer to the question, "What is the new medicine for ataxia?" and fuels optimism for future treatments across various forms of this debilitating condition.

Originally developed as a novel glutamate modulator, troriluzole has seen varied results across different clinical trials. This article specifically addresses the question: **how effective is troriluzole?** by examining its performance in studies for spinocerebellar ataxia (SCA) and other neurological conditions.

Riluzole was the first drug approved by the FDA for the treatment of Amyotrophic Lateral Sclerosis (ALS) in 1995. However, a newer medication, troriluzole, was developed as an optimized prodrug of riluzole, aiming to improve its pharmacokinetic properties and side effect profile.

Glutamate is the most abundant excitatory neurotransmitter in the human body, but its dysregulation is linked to numerous neurodegenerative diseases [1.2.1]. So, what is the mechanism of action of troriluzole? This third-generation prodrug primarily works by modulating and normalizing synaptic glutamate levels [1.3.1, 1.2.2].