Results about: Friedreich's ataxia

An “ambulance” for the brain

Chemists at IRB Barcelona patent and present a shuttle capable of transporting molecules into the brain; this achievement could facilitate the treatment of diseases with no therapy available.

98% of drugs targeting the central nervous system are discarded because they are unable to cross the physical barrier that protects the brain.

The IRB Barcelona scientists, together with clinical teams, are now evaluating the potential of their shuttle for rare and orphan diseases.

International patient advocates partner to fund Spanish gene-therapy project to treat Friedreich's ataxia

Scientists at the Institute for Research in Biomedicine (IRB) and the “Centro de Biología Molecular Severo Ochoa” (CBMSO) launched the project two months ago.

The project is the result of an initiative of Spanish people affected by this rare disease who are grouped in GENEFA in collaboration with the Spanish Federation of Ataxias and the BabelFAmily. The Friedreich’s Ataxia Research Alliance (FARA), one of the main patients’ associations in the United States now joins the endeavour.

The support provided by FARA will account for 50% of the project budget of 300,000 euros. Of note, FARA’s funds come from the support of patient families and communities raising funds at a grassroots level and from other International advocates such as FARA – Ireland.