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The investigational therapy DT-216 safely worked to more than double the activity of the FXN gene — involved in energy production in cells — in people with Friedreich’s ataxia (FA). That’s according to new data from a single-ascending dose (SAD) Phase 1 clinical trial, which showed the treatment candidate was…

A $4.8 million grant from the California Institute for Regenerative Medicine (CIRM) will support a research project hoping to move a one-time gene editing treatment for Friedreich’s ataxia closer to clinical trials. “If successful, this approach could prevent the neurologic and cardiac complications in [Friedreich’s ataxia] and address the pressing…

People with Friedreich’s ataxia (FA) who also have diabetes, a condition that causes their blood glucose (sugar) level to become too high, are more likely than those who don’t have diabetes to have worse outcomes from COVID-19, according to a single-center study. Having diabetes increased both the chance of…

Low levels of the frataxin protein — the underlying cause of Friedreich’s ataxia (FA) — and the ensuing problems in mitochondria, the cells’ powerhouses, may activate certain immune responses in the disease, a study suggests. In lab-grown human heart, muscle precursor, and connective tissue cells, suppressing frataxin production was…

A four-week Phase 2 clinical trial that’ll be testing CTI-1601, Larimar Therapeutics’ experimental therapy for Friedreich’s ataxia (FA), is now enrolling in the U.S., the company announced in a press release. Patient recruitment began shortly after the U.S. Food and Drug Administration (FDA) lifted its full hold…

The Phase 1 clinical program of DT-216, Design Therapeutics’ experimental therapy for Friedreich’s ataxia (FA), remains on track, with initial data expected by year’s end. The program includes a single-ascending dose Phase 1a trial (NCT05285540) and a multiple-ascending dose Phase 1b study (NCT05573698), both testing the…

Larimar Therapeutics has received a patent relative to CTI-1601, its experimental and potentially disease-modifying treatment for Friedreich’s ataxia (FA), the company announced. The patent, No. 11,459,363 and titled “Materials and Methods for Treating Friedreich’s Ataxia,” provides protection for composition of matter relative to CTI-1601 until at least July 2040.

In a reversal, the U.S. Food and Drug Administration (FDA) has informed Reata Pharmaceuticals that it will not hold an advisory committee meeting to discuss the company’s application for omaveloxolone, its oral treatment candidate for Friedreich’s ataxia. The FDA’s reversal means that patients and the FA community will…

A mutation in the gene SIRT6 is associated with less severe neurological symptoms and visual impairment among people with Friedreich’s ataxia, a new study reports. “We have identified a [mutation] in SIRT6 as a potential modifier of [Friedreich’s ataxia] neurological features,” researchers wrote. “The less common CT genotype…

A new mouse model called YG8-800, which accurately captures key features of Friedreich’s ataxia such as neurological dysfunction and heart disease, could be a useful tool for studying genetic therapies for the disease. That’s according to the study, “A promising mouse model for Friedreich Ataxia progressing…