2 documents found, page 1 of 1
Long-term efficacy and safety of inotersen for hereditary transthyretin amyloid...
Brannagan, Thomas H.; Coelho, Teresa; Wang, Annabel K.; Polydefkis, Michael J.; Dyck, Peter J.; Berk, John L.; Drachman, Brian; Gorevic, PeterBackground: Hereditary transthyretin amyloidosis (hATTR/ATTRv) results from the deposition of misfolded transthyretin (TTR) throughout the body, including peripheral nerves. Inotersen, an antisense oligonucleotide inhibitor of hepatic TTR production, demonstrated a favorable efficacy and safety profile in patients with the polyneuropathy associated with hATTR in the NEURO-TTR (NCT01737398) study. We report long...
Transthyretin deposition in the eye in the era of effective therapy for heredit...
Buxbaum, Joel N.; Brannagan, Thomas; Buades-Reinés, Juan; Cisneros, Eugenia; Conceição, isabel; Kyriakides, Theodoros; Merlini, Giampaolo; Obici, LauraBackground: Ocular abnormalities have been known to occur in hereditary amyloidotic polyneuropathy since the 1950s. While vitreous opacities and scalloped pupils were described early it has become evident that every component of the eye from the conjunctiva to the retinal vasculature can be involved. Reports from the major centres in Japan, Portugal and Sweden, which primarily treat patients with ATTRV30M, have...