A FATHER-of-two from Thornbury will tackle a strenuous 220-mile cycle across rocky terrain for two sisters whose rare genetic disorder leads them to jerk uncontrollablyPhil Lucker, 36, will take on the Escape the Dragon challenge, riding from Caernarfon in Wales back to Thornbury in three days bet More …

While Huntington's disease (HD) is currently incurable, the HD research community anticipates that new disease-modifying therapies in development may slow or minimize disease progression. The success of HD research depends upon the identification of reliable and sensitive biomarkers to track "Char More …

LEXINGTON, Mass. - Synageva BioPharma Corp. (Synageva), a biopharmaceutical company developing therapeutic products for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to sebelipase alfa for the treatment of early onset Accor More …