News

Heart function OK with high dose of Skyclarys in study

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…

Friedreich’s ataxia mutations disrupted with gene editing

A new gene editing technique disrupted the mutations that cause Friedreich’s ataxia (FA) in a mouse model, a study reports. A type of base editing, the technique swaps one genetic building block, or nucleotide, for another. These edits can interrupt long repetitions of three-letter DNA sequences (trinucleotide repeats, or…

Good safety seen with FA drug Skyclarys over 1 year in real world

The approved Friedreich’s ataxia (FA) drug Skyclarys (omaveloxolone) was found to be generally safe and well tolerated in people with FA in the real world, according to a new U.S. study that tested the medication’s safety over one year. Most patients remained on Skyclarys for more than 12 months…

Scientists design molecules that target genetic defects in FA

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…

Nutrition monitoring may help patients keep healthy weight: Study

Children with Friedreich’s ataxia are often underweight and short for their age, while adult patients are frequently overweight, so regular nutrition checks are important for maintaining healthy weight, a study from Europe suggested. The study, “Longitudinal analysis of anthropometric measures over 5 years in patients with Friedreich ataxia in…

Larimar to seek nomlabofusp’s accelerated approval this year

Larimar Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) this year to grant accelerated approval to nomlabofusp, its experimental treatment for Friedreich’s ataxia (FA). “The strong clinical and regulatory progress across our nomlabofusp program reinforces the timing of our planned Biologics License Application (BLA)…