A new study outlines the design and rationale for APOLLO, a Phase 3 clinical trial (NCT02510261) to evaluate the effect of patisiran (ALN-TTR02) in…
News
HATTR-PN
NewsImaging Is as Good as Tissue Sampling at Detecting Amyloid Deposits in Some Organs, Study Finds
Body-wide imaging can detect amyloid protein deposits in the organs of people with familial amyloid polyneuropathy (FAP), making it possible to assess the effects…
Researchers presented the main features of primary amyloidosis, showing the need for careful diagnosis in patients that present with neuropathic symptoms, especially autonomic neuropathy, to…
HATTR-PN
NewsTafamidis Delays Disease Progression in Patients with Transthyretin Amyloid Polyneuropathy, Pfizer Study Suggests
An interim analysis of the Phase 3 trial for tafamidis (Fx-1006A) as a therapy for patients with hereditary transthyretin amyloid polyneuropathy (TTR-FAP) shows the…
HATTR-PN
NewsDisease Knowledge in People With FAP Family History Differs From Those With Treatable Diseases
People at risk for familial amyloid polyneuropathy (FAP) caused by mutations in the TTR gene have different levels of knowledge about their disease than people…
HATTR-PN
NewsRare TTR Mutation Seen to Cause FAP in Chinese Family
Chinese researchers report the case of a family affected by familial amyloid polyneuropathy (FAP) caused by a less common mutation in the transthyretin…
Patisiran (ALN-TTR02) led to an improvement in average nerve dysfunction in 26 patients with familial amyloid polyneuropathy (FAP), a rare disease that causes organ or…
GlaxoSmithKline (GSK) has backed away from the chance to license two Ionis Pharmaceuticals treatments for familial amyloid polyneuropathy (FAP) — also known as TTR…
A new clinical trial called TRAM aims to determine the prevalence of genetic mutations in the transthyretin (Ttr) gene, the underlying cause of TTR familial…
HATTR-PN
NewsMIBG Scans Prove Valuable in Determining Familial Amyloid Polyneuropathy Patients’ Outcomes, Study Shows
An imaging technique that uses tiny amounts of radioactive chemicals can help doctors predict the outcomes of cases of familial amyloid polyneuropathy, according to a Portuguese…
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