News

The COVID-19 pandemic will delay development work on a potential gene therapy, and the start of a planned and possibly pivotal clinical trial in the small molecule treatment PTC743, both intended for people with Frederich’s ataxia (FA), PTC Therapeutics said in a quarterly report. PTC now expects to start…

Scientists discovered two microRNAs — tiny RNA molecules that control the activity of several genes — playing a key role in the development of Friedreich’s ataxia (FA) that may be promising candidates for its treatment. Their findings were reported in the study, “A Comprehensive Transcriptome…

FFF Enterprises and Bionews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone. Recognized as the nation’s leading supplier of…

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…

Altered regulation of a protein called actin may contribute to neurodegeneration in Friedreich’s ataxia, according to a study in a mouse model of the disease. The work highlighted a potential association between the underlying cause of FA (lack of the frataxin protein) and both over-activation of…

Eye health correlates with neurological disability in people with Friedreich’s ataxia (FA), a clinical study reports. These findings support the potential of specific measures of vision and eye health to evaluate disease progression in this patient population, the scientists said. The study “Ocular Involvement in Friedreich Ataxia Patients and…

The COVID-19 pandemic has not affected the preclinical program of XCUR-FXN, an experimental treatment for Friedreich’s ataxia (FA), Illinois-based Exicure said. Since biotechnology companies were designated essential businesses by the state’s governor J.B. Pritzker, Exicure will continue its research programs despite the the “stay-at-home” order in…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

To minimize risk during the COVID-19 pandemic, Reata Pharmaceuticals is implementing at-home visits to deliver omaveloxolone to Friedreich’s ataxia (FA) patients and to assess the treatment’s safety in an extension study of the MOXIe Phase 2 clinical trial. “The COVID-19 pandemic presents an unprecedented threat to public…