The results showed that before 35 years of age, leukocyte telomeres were longer in patients than in controls, whereas the reverse applies in patients above 36 years of age. Interestingly, LTL was greater than controls at any age in heterozygous subjects. Overall, the present study indicates that telomere length analysis in FRDA may be a relevant biomarker for following the stages of the disease.
Monday, September 9, 2024
Unusual Age-Dependent Behavior of Leukocytes Telomere Length in Friedreich's Ataxia
Scarabino D, Veneziano L, Nethisinghe S, Mantuano E, Fiore A, Granata G, Solanky N, Zanni G, Cavalcanti F, Corbo RM, Giunti P. Unusual Age-Dependent Behavior of Leukocytes Telomere Length in Friedreich's Ataxia. Mov Disord. 2024 Sep 5. doi: 10.1002/mds.29976. Epub ahead of print. PMID: 39235665.
A Pilot Phase 2 Randomized Trial to Evaluate the Safety and Potential Efficacy of Etravirine in Friedreich Ataxia Patients
Paparella, G.; Stragà, C.; Pesenti, N.; Dal Molin, V.; Martorel, G.A.; Merotto, V.; Genova, C.; Piazza, A.; Piccoli, G.; Panzeri, E.; et al. A Pilot Phase 2 Randomized Trial to Evaluate the Safety and Potential Efficacy of Etravirine in Friedreich Ataxia Patients. Children 2024, 11, 958, doi:10.3390/children11080958.
Etravirine was well tolerated. Etravirine completely stopped the progression of the SARA score during the 4-months treatment period, compared to the 4 months pre and post treatment. It increased peak workload, while the improvement of peak oxygen uptake was not statistically significant. No changes in the cardiac measures were observed. Health and QoL measures showed a worsening at the suspension of the drug. In this open trial etravirine significantly improved neurological function and was generally safe and reasonably tolerated. This suggests that etravirine represents a potential therapeutic agent in FRDA deserving testing in a randomized placebo controlled clinical trial.
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