Diseases, Conditions, Syndromes

Vitamin B3 treatment for ataxia shows promise in first human trial

(Medical Xpress)—A form of vitamin B3 has shown early promise against Friedreich's ataxia, a debilitating degenerative disease with no treatment or cure, in the first human trial of the treatment involving UCL researchers.

Medical research

Friedreich's ataxia: An effective gene therapy in an animal model

The team led by Hélène Puccio, director of research for Inserm at the Institute of Genetics and Molecular and Cellular Biology in close collaboration with Patrick Aubourg's team has demonstrated, in the mice, the efficacy ...

Diseases, Conditions, Syndromes

What is the cost of rare diseases such as Friedreich's Ataxia?

What is the cost of rare diseases such as Friedreich's Ataxia? By analyzing direct and indirect costs of care research in BioMed Central's open access journal Orphanet Journal of Rare Diseases calculated that conservatively ...