ALLMedicine™ Friedreich Ataxia Center

Jun 1st, 2022 - During the double-blind, placebo-controlled phase, participants will be stratified by baseline mFARS score (<40 versus ≥40), age of disease onset (<14 versus ≥14), and age at screening (≤21 years or >21 years) and randomized to receive either vati...

May 21st, 2022 - Friedreich ataxia is a rare autosomal recessive, neuromuscular degenerative disease caused by an expansion of a trinucleotide [guanine-adenine-adenine (GAA)] repeat in intron 1 of the FXN gene. It is common in the White population, characterized b...

May 18th, 2022 - Reference Range Vitamin E is a fat-soluble vitamin that acts as an antioxidant and free-radical scavenger in lipophilic environments. Vitamin E requires bile for absorption, and 25% of it is absorbed orally. The vitamin is stored in adipose tissue...

May 18th, 2022 - Vitamin E is a fat-soluble vitamin that acts as an antioxidant and free-radical scavenger in lipophilic environments. Vitamin E requires bile for absorption, and 25% of it is absorbed orally. The vitamin is stored in adipose tissue, liver, and mus...

May 18th, 2022 - Reference Range Vitamin E is a fat-soluble vitamin that acts as an antioxidant and free-radical scavenger in lipophilic environments. Vitamin E requires bile for absorption, and 25% of it is absorbed orally. The vitamin is stored in adipose tissue...

Jun 1st, 2022 - During the double-blind, placebo-controlled phase, participants will be stratified by baseline mFARS score (<40 versus ≥40), age of disease onset (<14 versus ≥14), and age at screening (≤21 years or >21 years) and randomized to receive either vati...

May 3rd, 2022 - Friedreich's ataxia (FA) is a rare autosomal recessive degenerative disorder characterized by ataxia, dysarthria, sensory loss, diabetes and cardiomyopathy. The discovery of the abnormal gene in FA and its product (frataxin) has provided insight i...

Apr 12th, 2022 - The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic and pharmacodynamic effects of intravenous DT-216 in adult patients with Friedreich Ataxia. This single ascending dose study is randomized, double-blind, placebo-co...

Mar 31st, 2022 - Friedreich ataxia is a rare progressive neurodegenerative disease presenting in childhood or late adolescence for which no treatment is currently available. The disease is caused by the defective synthesis of Frataxin, a mitochondrial protein synt...

Nov 18th, 2021 - Friedreich ataxia (FRDA) is the most common hereditary ataxia, with an estimated prevalence in Caucasians of 1 in 30,000. Neurological features of FRDA are progressive gait and limb ataxia, absent lower limb reflexes, and loss of position and vibr...

Jun 2nd, 2021 - Jordan Janz knew his gamble on an experimental gene therapy for his rare disease might be paying off when he returned to work and a friend sniffed him. "He said, 'you have a normal smell, you smell good,'" Janz recalled. "And I'm like, 'that's pro...

Aug 25th, 2010 - A Phase 3, Double-blind, Placebo-Controlled Trial of Idebenone in Friedreich Ataxia Lynch DR, Perlman SL, Meier T Arch Neurol. 2010;67:941-947 Summary The goal of this randomized, double-blind, placebo-controlled intervention trial was to evaluate...