Ionis and Alnylam square off at rare disease meeting in Paris

Today, doctors, patients and advocacy groups are gathered at the first annual European ATTR Amyloidosis meeting in Paris to hear clinical developments in the treatment of a family of rare disorders.

​ATTR amyloidosis is a group of multisystem diseases with poor prognoses, in which abnormal amyloid proteins accumulate and damage body organs and tissues.

Other than liver transplantation, the only approved therapy is Pfizer’s (NYSE: PFE) Vyndaqel (tafamidis), which has been prescribed in Europe for several years but has yet to be approved in the USA.