Updates

January 15, 2019January 15, 2019
Longer ATXN2 Coding Sequence Linked to Earlier TTR-FAP Onset in Val30Met Families, Study Shows

Length of naturally occurring repeats in the sequence of the ATXN2 gene may affect the age of onset of transthyretin (TTR)-related familial amyloid polyneuropathy (FAP). ... Read more
January 8, 2019January 8, 2019
Vyndaqel May Help Halt Neurological Disease Progression in Patients With Val30Met Mutation, Study Reveals

Early diagnosis and prompt treatment with Vyndaqel (tafamidis) may help halt neurological disease progression in familial amyloid polyneuropathy (FAP) patients who are positive for the ... Read more
December 21, 2018December 21, 2018
Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of ... Read more
December 20, 2018December 20, 2018
FAP Patients with Val30Met Mutation Exhibit Differences in Japan, Study Finds

Familial amyloid polyneuropathy patients with the Val30Met mutation differ in clinical symptoms, gene variants, and age of disease onset between endemic (where the disease ... Read more
December 13, 2018December 13, 2018
Patients in France with FAP, Upper Limb Nerve Damage Often Misdiagnosed, Study Suggests

Familial amyloid polyneuropathy (FAP) with arm (upper limb) involvement is common in France and frequently missed because of incorrect diagnosis, a French study reported. This ... Read more

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News

FAP Diagnosis Worth Considering in Patients with Neuropathy and Cardiac and Eye Issues, Case Report Shows
December 4, 2018December 4, 2018
A woman diagnosed with late-onset familial amyloid polyneuropathy (FAP) showed severe sensory and motor ...
News

Researchers Report First Case of FAP with Chronic Dry Cough in Large Chinese Family
November 29, 2018November 29, 2018
Researchers have reported the first case of familial amyloid polyneuropathy (FAP) with chronic dry ...
News

Liver Transplant, Vyndaqel Prolong Life of Early-stage FAP Patients, Study Reports
November 15, 2018November 15, 2018
Liver transplant or therapy with Pfizer‘s Vyndaqel (tafamidis) can substantially extend the lives of people ...
News

Val30Met FAP Patients’ Loss of Sensation Occurs in Both Upper and Lower Limbs, Study Reports
November 6, 2018November 6, 2018
Familial amyloid polyneuropathy (FAP) patients who carry the Val30Met mutation experience loss of sensation (numbness), ...
News

Lengthening of Amyloid Fibrils May Lead to Nerve Cell Loss in FAP Patients, Study Suggests
November 1, 2018November 1, 2018
The distortion and subsequent atrophy of cells in the peripheral nervous system, due to ...
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 ...

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 ...
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 ...
Tegsedi Approved in US for Treatment of FAP in Adults
October 15, 2018October 15, 2018
Tegsedi Now Approved in Canada for FAP Treatment in Adults
October 11, 2018October 11, 2018
Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments
October 9, 2018October 9, 2018
Alnylam Submits New Drug Application in Japan for Patisiran in Treatment of Hereditary ATTR Amyloidosis
October 8, 2018October 8, 2018

#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
Portugal has the highest prevalence of transthyretin familial amyloid polyneuropathy (TTR-FAP) to date, although ...
3D Structure of TTR Protein Determines Its Cellular Effects, Study Reports
August 30, 2018August 30, 2018
Blood Vessel Response to Vasodilator Agent Can Help Determine FAP Severity, Study Suggests
August 28, 2018August 28, 2018

News

Sudoscan and Neuropad Can Detect Disease Severity in FAP Patients, Study Suggests
August 23, 2018August 23, 2018
Sudoscan and Neuropad, tests that assess sweat gland function, are effective at diagnosing familial ...
News

Considerable Burden That ATTR Poses Affects Patients and Caregivers Alike, Study Finds
August 21, 2018August 21, 2018
The considerable burden of both living with transthyretin amyloidosis (ATTR) and caring for someone ...
News

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 ...
News

Sudoscan, Test of Sweat Gland Function, Reliable in Assessing Peripheral Neuropathies, Study Reports
August 7, 2018August 7, 2018
Evaluating the activity of sweat glands provides valuable information to assess peripheral nerve cell ...
News

Management of FAP Patients Needs to Include Family Therapy, Researchers Say
August 2, 2018August 2, 2018
The hereditary nature of familial amyloid polyneuropathy (FAP), along with the severity and unpredictability of ...
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 ...

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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