Rising levels of a protein called neurofilament light chain (NfL) in the blood may indicate nerve damage due to familial amyloid polyneuropathy (FAP) even before the disease manifests, a study in 65 people reports. These findings suggest that testing for NfL levels could be used to detect nerve damage…
hATTR-PN
Wainua (eplontersen) is able to slow disease progression and improve life quality for people with familial amyloid polyneuropathy (FAP) regardless of nutritional status, sex, or certain genetic factors. That’s according to new subgroup analyses from the Phase 3 NEURO-TTRansform clinical trial (NCT04136184), whose top-line data supported Wainua’s…
A new mutation in the TTR gene was identified as the cause of late-onset familial amyloid polyneuropathy (FAP) in an elderly man in Argentina, a small case study indicates. The case highlights “the importance of including FAP-TTR among early differential diagnosis in patients with presumably idiopathic [of uncertain cause]…
Tegsedi (inotersen) safely and effectively slowed the progression of polyneuropathy, or damage to multiple nerves, when taken for up to about six years for 10 adults with familial amyloid polyneuropathy (FAP) in Brazil, according to a real-world study. While the data add to results of Tegsedi clinical trials,…
The past month was challenging for my family. My wife and I were expecting the birth of our new child at any moment, according to my wife’s symptoms and her doctors’ words. But at the same time, my mother-in-law was experiencing pain related to peripheral neuropathy, which has resulted…
I’m happy. That’s the best way to describe my state at this stage of my life. When my husband, Aubrey, passed away on May 22, 2022, my whole world was stirred and uprooted. He’d been diagnosed with hereditary ATTR amyloidosis in 2013, and his disease journey took…
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 noninvasive test that measures axonal excitability, or the processes that underlie electrical impulses along nerve fibers, could distinguish between the early and later stages of familial amyloid polyneuropathy (FAP), a study reports. “Axonal excitability has utility to identify early and progressive nerve dysfunction in [FAP],” the researchers wrote…
Treatment with Amvuttra (vutrisiran) may ease heart damage in people with familial amyloid polyneuropathy (FAP), a new analysis of the HELIOS-A Phase 3 clinical trial suggests. “The totality of assessments in this exploratory analysis of the HELIOS-A study provides evidence of potential benefit of [Amvuttra] on cardiac [heart-related] manifestations…
Recent Posts
- A look at the emotional and psychological effects of diagnosis
- ATTR amyloidosis cases on the rise in the US as diagnostics improve
- Nerve ultrasound able to spot silent damage in hATTR, study finds
- Heart rhythm problems common in adults with FAP-causing mutation
- The emotional burden of receiving negative genetic test results