Long-term treatment with Onpattro (patisiran) is generally safe and results in a stabilization or lessening of neurologic impairments in people with familial amyloid polyneuropathy (FAP), according to a meta-analysis of published studies. Onpattro was used for a mean of 17.4 months (about 1.5 years) among the 503 patients in the…
People with familial amyloid polyneuropathy (FAP) and other forms of hereditary transthyretin amyloidosis (hATTR) have abnormal blood levels of two proteins, called growth differentiation factor-15 (GDF-15) and uromodulin (Umod), a small study reports. The preliminary findings from 16 patients in Italy suggest GDF-15 and Umod may serve as biomarkers of…
The clinical symptoms of hereditary transthyretin amyloidosis (hATTR) — a group of disorders that includes familial amyloid polyneuropathy (FAP) — are highly influenced by the type of mutation in the TTR gene a patient has, a review study concludes. The findings indicate that a clinical profile marked mainly by neurologic problems,…
A second, therapeutic dose of Intellia Therapeutics‘ experimental gene-editing therapy NTLA-2001 safely and effectively reduced levels of the harmful transthyretin (TTR) protein in people with familial amyloid polyneuropathy (FAP). That’s according to data from the three FAP patients first treated with a suboptimal, low dose of NTLA-2001 who…
Four years of treatment with Onpattro (patisiran) safely prevents disease progression for most familial amyloid polyneuropathy (FAP) patients, according to a retrospective real-world study in Italy. “These data are not very different from those obtained in the APOLLO trial,” which supported the therapy’s approvals worldwide, the researchers wrote. The…
Among familial amyloid polyneuropathy (FAP) patients carrying the most common FAP-causing mutation Val30Met, treatment with tafamidis meglumine is most effective when given at the very early stages of the disease. That’s according to the findings of a real-world study in Spain, which linked a cutoff value of 15 or…
A few weeks after its approval in the U.S., Wainua (eplontersen) will now be available to adults with familial amyloid polyneuropathy (FAP). Orsini was selected as the exclusive specialty pharmacy partner to provide Wainua, which was co-developed by Ionis Pharmaceuticals and AstraZeneca. The medication will also be made available through integrated…
An ultrasound of the peripheral nerves could help monitor disease progression in people with early-stage familial amyloid polyneuropathy (FAP) who carry V30M, the most common disease-causing mutation. That’s according to a study in Sweden that also showed that while about half of patients analyzed showed no signs of peripheral…
Intellia Therapeutics is launching a pivotal Phase 3 clinical trial in the U.S. to evaluate the safety and effectiveness of its investigational gene-editing therapy NTLA-2001 in people with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study, which is expected to start by the end of the year, follows the recent…
A 63-year-old man in Japan with a large bulge in his right arm bicep, called “Popeye sign,” was diagnosed with late-onset familial amyloid polyneuropathy (FAP), according to a case report which suggests this sign has potential to diagnose the disease. The reason behind the symptom remains unclear in this…