Exciting results in drug trial for rare disease Friedreich’s ataxia – University College London Hospitals

A drug company has announced positive results in the first global trial of a drug for a rare neuromuscular disease Friedreich’s ataxia (FA) which included patients from the UCLH Ataxia Centre for the UK.

Reata Pharmaceuticals said FA patients who took omaveloxolone (Omav) for 48 weeks in a phase 2 trial named MOXIe had a statistically significant improvement in their symptoms compared to those receiving a placebo.

The company said the drug has the potential to be the first approved treatment for FA. It said that, subject to discussions with regulators, it now plans to apply for approval to market the drug internationally.

Click here to read full article https://www.uclh.nhs.uk/News/Pages/ExcitingresultsindrugtrialforrarediseaseFriedreichsataxia.aspx

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