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In this section, you will find the most recent FA research publications, many of which are funded by FARA, as well as information on upcoming conferences and symposiums. You can search for articles by date using the archive box in the right hand column. To locate FARA Funded or Supported Research, click the hyperlink in the right hand column. You may also search for specific content using key words or phrases in the search button at the top right of your screen. Please be sure to visit other key research sections of our website for information on FARA’s Grant Program and the Treatment Pipeline.
What the reader will gain: This review covers the most relevant aspects related with the therapeutic use of CoQ10, including existing formulations and their effects on its bioavailability.
Take home message: CoQ10 does not cause serious adverse effects in humans and new formulations have been developed that increase CoQ10 absorption. Oral CoQ10 is a viable antioxidant strategy in many diseases, providing a significant to mild symptomatic benefit. Idebenone and MitoQ are promising substitutive CoQ10-related drugs which are well tolerated and safe.Read More: Therapeutic use of coenzyme Q(10) and coenzyme Q(10)-related compounds and formulations
Idebenone has been used as therapy for Friedreich ataxia for more than a decade. Although several studies have assessed the influence of therapy on neurologic or cardiac function, there is a paucity of data surrounding patient-reported outcome measures. In an observational study of the effect of intermediate-dose idebenone (20 mg/kg per day) on quality of life and neurologic function measures, seven patients with Friedreich ataxia were assessed using the Pediatric Quality of Life Inventory, the International Cooperative Ataxia Rating Scale, and an Activities of Daily Living Scale before initiation of idebenone therapy and after 1 year of therapy.
The use of health-related quality of life scales as outcome measures in clinical trials is increasing. Although such measures have been validated in adults with Friedreich ataxia, they have not been studied in children with this disorder. The health-related quality of life of children with Friedreich ataxia was assessed using the PedsQL 4.0 Generic Core Module and Multidimensional Fatigue Scale. The scores from the Friedreich ataxia cohort were compared to those of control groups (children without a chronic disease).