Global Phase 3 BRAVE study will evaluate the efficacy and safety of omaveloxolone in children 2 to BRAVE study will explore the potential of omaveloxolone to address the critical unmet need of the ped ...

Global Phase 3 BRAVE study will evaluate the efficacy and safety of omaveloxolone in children 2 to <16 years old with ...

Ataxia can have a profound impact on an individual’s ability to perform their job duties—even with appropriate treatment.. For Canadians who can’t work due to this debilitating condition ...

Melissa Beiner, M.D., SCA Clinical Development Lead at Biohaven, commented, Our NDA filing is the culmination of over 8 years of clinical research a ...

The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for troriluzole for the treatment of adults with spinocerebellar ataxia (SCA).

Solid Biosciences announces FDA clearance for SGT-212, targeting Friedreich’s ataxia with dual gene therapy administration routes, initiating Phase 1b trial in 2025.

This model reproduces the phenomena common to many cerebellar neurodegenerative conditions, which is very important for testing pharmacological developments in this scientific area. Treatment of mice ...

Cerebellar abnormalities are the main cause of ataxia and likely worsen over the course of the disease. Nonetheless, there are limited symptomatic treatments available for cerebellar features.

They urged for increased use of early genetic and biochemical testing for Friedreich ataxia as a way to decrease treatment delays. Characterized by progressive neurodegeneration and multisystem ...

Car8 wdl/wdl mice also exhibit ataxia and dystonia, which are not normalized by propranolol (Jiao et al., 2005; White et al., 2016a; Miterko et al., 2021; Zhou et al., 2022). We found that, before ...