Posts by: Kara Elam

News

A case study of a Freidreich’s ataxia (FA) patient with associated cardiomyopathy reported that an experimental treatment — interferon gamma (INFγ) therapy — could improve cardiac function. The study, “Interferon gamma may improve cardiac function in Friedreich’s ... Read more

January 5, 2016January 5, 2016

FA Study Finds Frataxin Levels in Cells Unaffected by Interventions

News

Pfizer has published a study with potentially important implications for future therapeutic targets to treat Friedreich’s ataxia (FA). The study, titled “Perturbation of cellular proteostasis networks identifies pathways that modulate precursor ... Read more

December 9, 2015December 9, 2015

In Friedreich’s Ataxia Patient, Mutation Affected Proteins’ Ability to Fold

News

Researchers have shown that a potential new therapeutic target for patients with Friedreich’s ataxia (FA) could be a mutation that causes misfolding in specific proteins, which results in lowered protein ... Read more

August 6, 2015August 11, 2015

Robotic Devices Give Accurate Clinical Assessments for Patients With Friedreich’s Ataxia

News

A collaborative team of international researchers led by the Don Carlo Gnocchi Onlus Foundation, in Milan, Italy have shown that outcomes measured using robotic devices can improve the sensitivity of ... Read more

June 25, 2015July 2, 2015

BioBlast Presents Preclinical Proofs of Concept at Two Leading Scientific Meetings

News

BioBlast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage orphan disease-focused biotechnology company whose mission is to transform excellent science into safe and effective therapies for rare genetic diseases, has recently presented ... Read more

March 2, 2015March 2, 2015

National Ataxia Foundation Funds Study Investigating Spinocerebellar Ataxia Type 6

Friedreich's Ataxia, News

The National Ataxia Foundation (NAF), an organization dedicated to improving the lives of those affected by ataxia through support, education, and research, recently released a list of study proposals awarded funding for ... Read more