More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars…
News
The latest results of the Phase 3 NEURO-TTR study continue to demonstrate the therapeutic potential of inotersen for patients with hereditary TTR amyloidosis (hATTR)…
News
#NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says
As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives…
Advancing the study of rare diseases, including familial amyloid polyneuropathy (FAP), is the goal of a new research institute to be founded at the…
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…
Recent Posts
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