Updates

December 4, 2018December 4, 2018
FAP Diagnosis Worth Considering in Patients with Neuropathy and Cardiac and Eye Issues, Case Report Shows

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 ... Read more
November 29, 2018November 29, 2018
Researchers Report First Case of FAP with Chronic Dry Cough in Large Chinese Family

Researchers have reported the first case of familial amyloid polyneuropathy (FAP) with chronic dry cough — caused by a less common mutation — in ... Read more
November 15, 2018November 15, 2018
Liver Transplant, Vyndaqel Prolong Life of Early-stage FAP Patients, Study Reports

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, ... Read more
November 6, 2018November 6, 2018
Val30Met FAP Patients’ Loss of Sensation Occurs in Both Upper and Lower Limbs, Study Reports

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 ... Read more
November 1, 2018November 1, 2018
Lengthening of Amyloid Fibrils May Lead to Nerve Cell Loss in FAP Patients, Study Suggests

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

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News

#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach
October 30, 2018
With the U.S. midterm elections now less than two weeks away, patient advocacy groups ...
News

FAP Not Common in Patients with Charcot-Marie-Tooth Disease Type 2, Study Reports
October 23, 2018October 23, 2018
Lack of familial amyloid polyneuropathy (FAP)-causing mutations in patients with Charcot-Marie-Tooth disease type 2 ...
News

Tegsedi and Onpattro May Ably Treat FAP, But Are Overpriced, ICER Reports
October 19, 2018October 19, 2018
Prices for Tegsedi (inotersen) and Onpattro (patisiran) far exceed thresholds for cost-effectiveness, despite their ...
News

Tegsedi Approved in US for Treatment of FAP in Adults
October 15, 2018October 15, 2018
The U.S. Food and Drug Administration has granted approval to Tegsedi (inotersen), by Akcea Therapeutics and ...
News

Tegsedi Now Approved in Canada for FAP Treatment in Adults
October 11, 2018October 11, 2018
Tegsedi (inotersen), by Akcea Therapeutics and Ionis Pharmaceuticals, has been granted approval in Canada as a treatment ...
News

Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments
October 9, 2018October 9, 2018
Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced ...

Alnylam Submits New Drug Application in Japan for Patisiran in Treatment of Hereditary ATTR Amyloidosis
October 8, 2018October 8, 2018
Alnylam Pharmaceuticals announced the submission of a New Drug Application (NDA) to Japan’s Pharmaceuticals and Medical ...
#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington
September 21, 2018
More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to ...
September 20, 2018September 20, 2018
Rare Case of FAP and Mitochondrial Disease Reported in Study
September 18, 2018September 18, 2018
Somatostatin Analogues May Ease Chronic Diarrhea in FAP Patients, Pilot Study Reports
September 11, 2018September 11, 2018
Portugal Has Highest Prevalence of TTR-FAP, but Rate of New Cases Decreasing, Study Finds
September 4, 2018September 4, 2018

3D Structure of TTR Protein Determines Its Cellular Effects, Study Reports
August 30, 2018August 30, 2018
The effect of the familial amyloid polyneuropathy (FAP) hallmark protein transthyretin (TTR) on cells ...
Blood Vessel Response to Vasodilator Agent Can Help Determine FAP Severity, Study Suggests
August 28, 2018August 28, 2018
Sudoscan and Neuropad Can Detect Disease Severity in FAP Patients, Study Suggests
August 23, 2018August 23, 2018
Considerable Burden That ATTR Poses Affects Patients and Caregivers Alike, Study Finds
August 21, 2018August 21, 2018

FDA Approves Alnylam’s Onpattro (Patisiran) as FAP Therapy
August 14, 2018August 14, 2018
The U.S. Food and Drug Administration (FDA) has approved Alnylam Pharmaceuticals’ Onpattro (patisiran) as the ...
Sudoscan, Test of Sweat Gland Function, Reliable in Assessing Peripheral Neuropathies, Study Reports
August 7, 2018August 7, 2018
Management of FAP Patients Needs to Include Family Therapy, Researchers Say
August 2, 2018August 2, 2018

News

Vyndaqel May Ease CNS and Eye Manifestions in Certain FAP Patients, Study Suggests
July 31, 2018July 31, 2018
Treatment with 20 mg a day of oral Vyndaqel (tafamidis) is enough for the ...
News

11-Gene Analysis Based on Next-Gen Sequencing Could Improve FAP Diagnosis, Researchers Suggest
July 27, 2018July 27, 2018
Next-generation sequencing to analyze a panel of 11 specific genes may facilitate the diagnosis ...
News

Additional Data from APOLLO Trial Supports Patisiran as Promising FAP Therapy
July 24, 2018July 24, 2018
Alnylam Pharmaceuticals’ investigative patisiran treatment improves overall health and prevents worsening of walking ability ...
News

Non-Val30Met FAP Patients Exhibit Cardiac and Blood Vessel Dysfunction, Study Shows
July 19, 2018July 19, 2018
Familial amyloid polyneuropathy (FAP) patients who do not have a Val30Met mutation exhibit prevalent ...
News

Tegsedi Found to Improve Neuropathy Symptoms, Quality of Life in FAP Patients in Phase 3 Trial
July 17, 2018July 17, 2018
Treatment with the investigative therapy Tegsedi (inotersen) provides early and significant improvements in neuropathy symptoms ...
News

Tegsedi Approved in EU to Treat FAP Patients with Stage 1 or 2 Polyneuropathy
July 12, 2018July 12, 2018
The European Commission (EC) has approved Tegsedi (inotersen), by Akcea Therapeutics and Ionis Pharmaceuticals, to treat stage ...

September 6, 2017September 6, 2017

Tafamidis Delays Disease Progression in Patients with Transthyretin Amyloid Polyneuropathy, Pfizer Study Suggests

News

An interim analysis of the Phase 3 trial for tafamidis (Fx-1006A) as a therapy for patients with hereditary transthyretin amyloid polyneuropathy (TTR-FAP) shows the investigational treatment delays disease progression, Pfizer announced. The study, “Long-term safety and efficacy of tafamidis for ... Read more

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