Safety and efficacy of omaveloxolone v/s placebo for the treatment of Friedreich's ataxia in patients aged more than 16 years: a systematic review

Umrao, A., Pahuja, M. & Chatterjee, N.S. Safety and efficacy of omaveloxolone v/s placebo for the treatment of Friedreich's ataxia in patients aged more than 16 years: a systematic review. Orphanet J Rare Dis 19, 495 (2024). doi:10.1186/s13023-024-03474-6 

The results of the Omav treatment indicated a persistent benefit to FA patients who received Omav for an extended period. The greater effect on longer GAA lengths than shorter repeats and the lower degree of improvement in pes cavus patients suggest that Omav aims for the most severe or progressive abnormalities in FA patients in a time-dependent manner.

Therefore, based on the available literature and the quality of the studies, further randomized controlled trials may include patients with progressive disease with the consideration of pes cavus, foot deformity. Considering the progressive nature of the disease, long-term follow-up beyond three years to constantly evaluate the effect of Omav on FA patients shall be considered.

Health-Related Quality of Life in Patients with Friedreich Ataxia Using Mobility Assistive Technologies: Limited Fit of the EQ-5D-3L Mobility Dimension

Health-Related Quality of Life in Patients with Friedreich Ataxia Using Mobility Assistive Technologies: Limited Fit of the EQ-5D-3L Mobility Dimension. Maresa Buchholz, Michelle Pfaff, Audrey Iskandar, Kathrin Reetz, Jörg B. Schulz, Marcus Grobe-Einsler, Thomas Klockgether, Bernhard Michalowsky, EFACTS Study Group, Neurology and Therapy, Print ISSN: 2193-8253, Elektronische ISSN: 2193-6536, DOI: 10.1007/s40120-024-00694-7 

There are concerns about using HRQoL measures, such as the EQ-5D, in mobility-impaired individuals who had difficulties finding appropriate response options for mobility-related items. Our findings demonstrated limitations of the EQ-5D-3L mobility item in patients with FA using MAT, detecting a noticeable amount of non-responses in the mobility item in wheelchair users and a tendency to the mid-response level “some problems” in individuals using walking aids.