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Gene Replacement and Editing

On October 8, 2022 By voicesoffa
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Significant advances in gene replacement therapy and the ability to edit genes have garnered much interest as a treatment approach

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Oligonucleotides

On October 7, 2022 By voicesoffa
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Nucleic acids are the building blocks of DNA and RNA. Oligonucleotides are chemically synthesized short DNA or RNA molecules that

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DT-216, Gene-TAC

On October 6, 2022 By voicesoffa
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Design Therapeutics is advancing a novel approach to activate transcription of the FXN gene and restore the production of frataxin

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Resveratrol

On October 5, 2022 By voicesoffa
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Resveratrol is a naturally occurring compound found in the skin of red grapes. Resveratrol has been under investigation as a

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Etravirine

On October 4, 2022 By voicesoffa
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Etravirine (Brand name: Intelence) – Dr. Roberto Testi and his team at the University of Rome and Fratagene recently reported

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CTI-1601 (TAT-Frataxin)

On October 3, 2022 By voicesoffa
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Dr. Mark Payne, who first described the possibility of frataxin replacement therapy via TAT-Frataxin, has been developing this exciting prospect

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Dimethyl Fumarate in Friedreich’s ataxia (FA)

On October 1, 2022 By voicesoffa
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Dr. Gino Cortopassi at the University of California Davis and other groups have observed that Nrf2 (Nuclear factor (erythroid-derived 2)-like

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Elamipretide

On September 30, 2022 By voicesoffa
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Elamipretide is a peptide compound developed by Stealth Therapeutics. It targets the inner mitochondrial membrane where it binds reversibly to

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PTC-743 Drug

On September 28, 2022 By voicesoffa
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PTC-743 / Vatiquinone (previously EPI-743), is a follow-on or “sister” compound to EPI-A0001, which aims to improve mitochondrial and cellular

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A systematic review of disease prevalence, health-related quality of life, and economic outcomes associated with Friedreich’s Ataxia

On September 27, 2022 By voicesoffa
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It is important to understand the burden of F A, including its extent and the most salient elements. The objective

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