The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business…
News
The protein cathepsin E — known for regulating immune system cells — may be a potential biomarker for familial amyloid polyneuropathy (FAP), a…
Diagnosing hereditary amyloid transthyretin (ATTRv) amyloidosis with polyneuropathy, also called familial amyloid polyneuropathy (FAP), is challenging, particularly in in places where the disease is…
News
Eye Disease Common in FAP Patients with Ser77Tyr Mutation, May Serve as Biomarker, Research Suggests
People with a type of FAP caused by a Ser77Tyr mutation are more likely to develop conjunctival lymphangiectasia, an uncommon clinical condition characterized…
Tegsedi (inotersen) has been approved in Brazil to treat stage 1 or 2 nerve damage (polyneuropathy) in adults with…
The annual costs and disease burden associated with hereditary transthyretin amyloidosis polyneuropathy (ATTRv-PN), also known as familial amyloid polyneuropathy (FAP), are considerable but within the…
Although uncommon, eye manifestations can affect patients with familial amyloid polyneuropathy (FAP). These are mostly caused by opacities in the jelly liquid of…
Two FDA-approved treatments for high blood pressure, diuretics called indapamide and hydrochlorothiazide, might also treat diseases associated with amyloid deposits, such as…
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to…
Alnylam Pharmaceuticals has opened its second annual competitive grants program, supporting patient advocacy projects worldwide that aim to address the unmet and diverse needs of…
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