Bernstein analyst Geoffrey Porges estimates there are probably about 3, 000 patients with aHUS in the U.S. and Europe.

FORBES: $500,000-A-Year Drug Is Bright Light For Pharma

In a mid-stage study, 13 of 15 patients who received Soliris did not require further treatment for aHUS. Based on some back-of-the-envelope math based on the incidence of aHUS in France and Italy, Sanford C.

FORBES: $500,000-A-Year Drug Is Bright Light For Pharma

Rachel Bennett from Exeter, whose son Felix was diagnosed with aHUS at six months, said Eculizumab was "incredible" and had given him a normal childhood for the first time at the age of nearly four.

BBC: Barnstaple kidney patient Elena Lilley's fight for drug