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October 11, 2018October 11, 2018

Tegsedi Now Approved in Canada for FAP Treatment in Adults

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Tegsedi (inotersen), by Akcea Therapeutics and Ionis Pharmaceuticals, has been granted approval in Canada as a treatment for stage 1 or stage 2 polyneuropathy in adults with hereditary transthyretin (hATTR) amyloidosis, also ... Read more

October 9, 2018October 9, 2018

Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

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Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based ... Read more

October 8, 2018October 8, 2018

Alnylam Submits New Drug Application in Japan for Patisiran in Treatment of Hereditary ATTR Amyloidosis

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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 treatment for hereditary transthyretin-mediated (hATTR) amyloidosis. ... Read more

September 21, 2018

#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

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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 Rare Diseases & Orphan Products ... Read more

September 20, 2018September 20, 2018

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Tests used to assess sensitivity to heat and cold may help diagnose patients with familial amyloid polyneuropathy (FAP) earlier as well as track their improvement after liver transplant, a ... Read more

September 18, 2018September 18, 2018

Rare Case of FAP and Mitochondrial Disease Reported in Study

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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 the journal Amyloid. The report ... Read more

September 11, 2018September 11, 2018

Somatostatin Analogues May Ease Chronic Diarrhea in FAP Patients, Pilot Study Reports

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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 suggests. The medicines, however, were linked to ... Read more

September 4, 2018September 4, 2018

Portugal Has Highest Prevalence of TTR-FAP, but Rate of New Cases Decreasing, Study Finds

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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 national epidemiological study shows. Cases ... Read more

August 30, 2018August 30, 2018

3D Structure of TTR Protein Determines Its Cellular Effects, Study Reports

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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 researchers. Their study, “A FTIR ... Read more

August 28, 2018August 28, 2018

Blood Vessel Response to Vasodilator Agent Can Help Determine FAP Severity, Study Suggests

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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 with familial amyloid polyneuropathy ... Read more

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