Alnylam Pharmaceuticals announced the submission of a New Drug Application (NDA) to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for the approval of patisiran as a…
News
More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD…
The rare case of a patient in Italy who had familial amyloid poyneuropathy (FAP) in addition to mitochondrial disease was described in a report published in…
Treatment with somatostatin analogues helps to ease the symptoms of difficult-to-treat diarrhea associated with familial amyloid polyneuropathy (FAP), a two-year pilot study…
Portugal has the highest prevalence of transthyretin familial amyloid polyneuropathy (TTR-FAP) to date, although the number of new cases is decreasing, data from a…
The effect of the familial amyloid polyneuropathy (FAP) hallmark protein transthyretin (TTR) on cells is associated with its specific three-dimensional shape, according to Italian…
A noninvasive evaluation of the response of skin blood vessels to a vasodilator compound can help detect the extent of nerve cell damage in patients…
Sudoscan and Neuropad, tests that assess sweat gland function, are effective at diagnosing familial amyloid polyneuropathy (FAP) and assessing small nerve fiber damage, according…
The considerable burden of both living with transthyretin amyloidosis (ATTR) and caring for someone with the disease affects the mental and physical health of patients…
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