An experimental gene therapy injected directly into the eye preserved the structure and function of the retina — the light-sensitive tissue at the back of the eye — in mouse models of Friedreich’s ataxia (FA), according to a proof-of-concept study. These data support “continued efforts toward the development of…
Leriglitazone, an oral therapy being investigated as a Friedreich’s ataxia (FA) treatment, extends nerve cell survival, improves mitochondrial function, helps balance iron levels, and prevents ferroptosis (a type of iron-dependent cell death), a cell-based study showed. Data also showed that combining leriglitazone with lower doses of the approved…
The investigational therapy nomlabofusp increased the levels of frataxin, the protein deficient in people with Friedreich’s ataxia (FA), in several tissues of animal models, and improved heart function and survival in an FA mouse model, a study showed. The findings are consistent with benefits of the Larimar…
Four years of daily treatment with Skyclarys (omaveloxolone) continues to slow the progression of Friedreich’s ataxia (FA) compared to what would be expected in patients without treatment. That’s according to new data from the extension study of the Phase 2 MOXIe clinical trial (NCT02255435), which also showed…
Supplementation with alpha-lipoic acid (ALA), a naturally occurring antioxidant, boosts the production of frataxin, the protein that’s deficient in people with Friedreich’s ataxia (FA), according to a cell-based study. Alpha-lipoic acid reversed many cellular features of FA, including dysfunction of energy-producing mitochondria, impaired iron metabolism, and oxidative stress, a…
A new study reports that Skyclarys (omaveloxolone) improves the heart’s ability to contract and pump blood while reducing inflammation and heart failure markers in a mouse model of Friedreich’s ataxia (FA) with severe heart disease. Skyclarys had no impact on the development of scar tissue, or fibrosis, in…
The U.S. Food and Drug Administration (FDA) has provided Larimar Therapeutics with clear expectations for an application seeking accelerated approval of nomlabofusp, its potentially disease-modifying therapy for Friedreich’s ataxia (FA), according to the drug developer. Accelerated approval is a type of conditional approval based on early clinical…
A high dose of the Friedreich’s ataxia (FA) treatment Skyclarys (omaveloxolone), far above the approved dose to match blood levels when the therapy is taken with food, did not affect heart function in healthy individuals, a study confirms. The study, “Effect of a Supratherapeutic Dose…
Scientists have designed molecules to correct the effect of DNA expanded repeats that cause Friedreich’s ataxia (FA), according to a new study. Using patient-derived cells, experiments demonstrated that these molecules, known as anti-gene oligonucleotides, or A-GOs, markedly boosted the production of frataxin, which is the protein that’s deficient in…
The U.S. Food and Drug Administration (FDA) granted priority review to PTC Therapeutics‘ application seeking the approval of vatiquinone, an oral treatment for children and adults with Friedreich’s ataxia (FA). The agency will announce a decision by Aug. 19, the company said. “We are excited to be…
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