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December 4, 2018December 4, 2018

FAP Diagnosis Worth Considering in Patients with Neuropathy and Cardiac and Eye Issues, Case Report Shows

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A woman diagnosed with late-onset familial amyloid polyneuropathy (FAP) showed severe sensory and motor neuropathy, or nerve damage, as well as eye and cardiac impairments, a case report describes. ... Read more

November 29, 2018November 29, 2018

Researchers Report First Case of FAP with Chronic Dry Cough in Large Chinese Family

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Researchers have reported the first case of familial amyloid polyneuropathy (FAP) with chronic dry cough — caused by a less common mutation — in a large Chinese family. The ... Read more

November 15, 2018November 15, 2018

Liver Transplant, Vyndaqel Prolong Life of Early-stage FAP Patients, Study Reports

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Liver transplant or therapy with Pfizer‘s Vyndaqel (tafamidis) can substantially extend the lives of people with familial amyloid polyneuropathy (FAP) in early stages of the disease, a Portuguese study has shown. ... Read more

November 6, 2018November 6, 2018

Val30Met FAP Patients’ Loss of Sensation Occurs in Both Upper and Lower Limbs, Study Reports

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Familial amyloid polyneuropathy (FAP) patients who carry the Val30Met mutation experience loss of sensation (numbness), especially sense of touch pressure, rather than heat pain or thermal sensibility, in both their ... Read more

November 1, 2018November 1, 2018

Lengthening of Amyloid Fibrils May Lead to Nerve Cell Loss in FAP Patients, Study Suggests

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The distortion and subsequent atrophy of cells in the peripheral nervous system, due to the accumulation and lengthening of amyloid fibrils, may be behind nerve cell loss in patients ... Read more

October 30, 2018

#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach

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With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Creditand ... Read more

October 23, 2018October 23, 2018

FAP Not Common in Patients with Charcot-Marie-Tooth Disease Type 2, Study Reports

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Lack of familial amyloid polyneuropathy (FAP)-causing mutations in patients with Charcot-Marie-Tooth disease type 2 (CMT2) means that genetic screening for FAP in this patient population is only required in ... Read more

October 19, 2018October 19, 2018

Tegsedi and Onpattro May Ably Treat FAP, But Are Overpriced, ICER Reports

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Prices for Tegsedi (inotersen) and Onpattro (patisiran) far exceed thresholds for cost-effectiveness, despite their likely health benefits to people with hereditary transthyretin amyloidosis (hATTR) and their families, a report by ... Read more

October 15, 2018October 15, 2018

Tegsedi Approved in US for Treatment of FAP in Adults

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The U.S. Food and Drug Administration has granted approval to Tegsedi (inotersen), by Akcea Therapeutics and Ionis Pharmaceuticals, for the treatment of adults with hereditary transthyretin-mediated amyloidosis (hATTR), also called familial amyloid polyneuropathy (FAP). Along ... Read more

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

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