Long-term treatment with Onpattro (patisiran) is generally safe and results in a stabilization or lessening of neurologic impairments in…
Articles by Patricia Inácio, PhD
People with familial amyloid polyneuropathy (FAP) and other forms of hereditary transthyretin amyloidosis (hATTR) have abnormal blood levels of two…
The clinical symptoms of hereditary transthyretin amyloidosis (hATTR) — a group of disorders that includes familial amyloid polyneuropathy (FAP) —…
A second, therapeutic dose of Intellia Therapeutics‘ experimental gene-editing therapy NTLA-2001 safely and effectively reduced levels of the harmful…
Four years of treatment with Onpattro (patisiran) safely prevents disease progression for most familial amyloid polyneuropathy (FAP) patients, according…
Among familial amyloid polyneuropathy (FAP) patients carrying the most common FAP-causing mutation Val30Met, treatment with tafamidis meglumine is most…
A few weeks after its approval in the U.S., Wainua (eplontersen) will now be available to adults with familial…
An ultrasound of the peripheral nerves could help monitor disease progression in people with early-stage familial amyloid polyneuropathy (FAP)…
Intellia Therapeutics is launching a pivotal Phase 3 clinical trial in the U.S. to evaluate the safety and effectiveness…
A 63-year-old man in Japan with a large bulge in his right arm bicep, called “Popeye sign,” was diagnosed with…