Advancing the study of rare diseases, including familial amyloid polyneuropathy (FAP), is the goal of a new research institute to be founded at the…
News
People with late-onset familial amyloid polyneuropathy (FAP) with the Val30Met mutation in their transthyretin gene commonly have heart and involuntary nerve abnormalities, according to…
Researchers reported a rare case of a patient with type 1 familiar amyloid polyneuropathy (FAP) who went to the emergency room with…
The discovery of serum proteins that are specific to FAC (familial amyloid cardiomyopathy) and FAP (familial amyloid polyneuropathy) may aid in the diagnosis and…
A liver, donated by a patient with familial amyloid polyneuropathy (FAP) caused by a Ser50Arg mutation in the transthyretin (TTR) gene, caused amyloidosis in the…
HATTR-PN
NewsFAP Patients Might Have New Treatment Before Year’s End, as Trial Data Show Patisiran’s Benefits
Patients with hereditary ATTR amyloidosis, also called familial amyloid polyneuropathy (FAP), might soon have a new medication to look forward to, as data from a…
HATTR-PN
NewsStudy Explains Phase 3 Clinical Trial Evaluating Alnylam’s Patisiran as Potential FAP Therapy
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…
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…
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