Results about: rare diseases

Rare and orphan but not abandoned by science

On Friday 6 March in a conference at the CCCB, researcher Xavier Salvatella will explain the excellent moment being experienced by research into rare diseases.

Salvatella will present the breakthrough in his lab regarding Kennedy’s disease, a rare muscular atrophy that affects 1 in 36,000 men. “In five years’ time we could have a candidate drug tested in cells in vitro.”

This is the first of four conferences in the “Science today for the medicine of the future”, proposed by IRB Barcelona to explain the close link between basic research and medicine of the future, held on the occasion of the 10th anniversary of the centre.

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.