A group of researchers compiled a database of small RNAs that circulate in the blood of Friedreich’s ataxia (FA)…
Articles by Patricia Inácio, PhD
As therapies for Friedreich’s ataxia seek to increase the amount of frataxin, whose shortage underlies the disease, scientists conducting…
Compounds similar to the chemical methylene blue, which has been shown to have therapeutic benefits in several diseases, were seen…
Retrotope has submitted the design of a pivotal clinical trial for its Friedreich’s ataxia therapy RT001 to the U.S. Food and Drug…
Results from a Phase 1/2 clinical study support the safety and tolerability of RT001, an investigational therapy for Friedreich’s ataxia.
Researchers found that the use of mobility devices is associated with a poorer quality of life in children with…
EURORDIS-Rare Diseases Europe teamed up with biotech Shire and software giant Microsoft to establish the Global Commission to…
Australian researchers say a short-term rehabilitation program improved the health and well-being of a small group of patients with Friedreich’s…
Administering small molecules that mimic factors naturally found in the brain to a mouse model of Friedreich’s ataxia increased levels of…
New viral vectors were found to effectively transfer a normal and protein-producing frataxin gene into major tissues affected by Friedreich’s ataxia —…