Pfizer’s Vyndaqel (tafamidis) can significantly delay the progression of familial amyloid polyneuropathy, regardless of its severity before treatment or which…
Articles by Alice Melao
Alnylam Pharmaceuticals’ lead investigational therapeutic patisiran, for the treatment of hereditary ATTR (hATTR) amyloidosis, also known as familial amyloid polyneuropathy…
Ionis Pharmaceuticals has asked the European Medicines Agency (EMA) to approve its lead drug candidate inotersen to treat hereditary TTR…
Familial amyloid polyneuropathy (FAP) is a progressive disorder that can severely impact the quality of life of patients, causing…
Extra copies of DNA in energy-producing cell components known as mitochondria play a role in the development of the Val30Met-mutation…
The latest results of the Phase 3 NEURO-TTR study continue to demonstrate the therapeutic potential of inotersen for patients…
Patients with familial amyloid polyneuropathy (FAP) are in a constant pro-inflammatory state, with increased levels of interleukin-6 (IL-6) driven by…
The investigative drug inotersen was shown to significantly reduce neurological progression and improve quality of life in patients with familial…