Updates

February 12, 2019February 12, 2019
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions ... Read more
February 5, 2019February 5, 2019
Eye Issues Still Prevalent in Val30Met FAP Patients After Liver Transplant, Study Shows

Eye problems due to transthyretin (TTR) deposition are more prevalent among patients who have had hereditary amyloidosis for longer periods of time and have ... Read more
February 1, 2019
FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases

The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— ... Read more
January 29, 2019January 29, 2019
Vyndaqel Is Safe, Effective for People with TTR-FAP, Review Study Suggests

Treatment with Vyndaqel (tafamidis) is a safe, non-invasive way to delay neurologic progression of transthyretin familial amyloid polyneuropathy (TTR-FAP), and preserves patients’ nutritional status ... Read more
January 22, 2019January 22, 2019
Heart Problems Are Top Cause of Death for FAP Patients Who Had Liver Transplant, Study Says

Cardiac problems are the leading cause of death among transthyretin familial amyloid polyneuropathy (ATTR, also called FAP) patients who have had a liver transplant, ... Read more

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News

Longer ATXN2 Coding Sequence Linked to Earlier TTR-FAP Onset in Val30Met Families, Study Shows
January 15, 2019January 15, 2019
Length of naturally occurring repeats in the sequence of the ATXN2 gene may affect the ...
News

Vyndaqel May Help Halt Neurological Disease Progression in Patients With Val30Met Mutation, Study Reveals
January 8, 2019January 8, 2019
Early diagnosis and prompt treatment with Vyndaqel (tafamidis) may help halt neurological disease progression in ...
News

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish
December 21, 2018December 21, 2018
Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures ...
News

FAP Patients with Val30Met Mutation Exhibit Differences in Japan, Study Finds
December 20, 2018December 20, 2018
Familial amyloid polyneuropathy patients with the Val30Met mutation differ in clinical symptoms, gene variants, ...
News

Patients in France with FAP, Upper Limb Nerve Damage Often Misdiagnosed, Study Suggests
December 13, 2018December 13, 2018
Familial amyloid polyneuropathy (FAP) with arm (upper limb) involvement is common in France and frequently ...
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 ...

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 ...
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 ...
Val30Met FAP Patients’ Loss of Sensation Occurs in Both Upper and Lower Limbs, Study Reports
November 6, 2018November 6, 2018
Lengthening of Amyloid Fibrils May Lead to Nerve Cell Loss in FAP Patients, Study Suggests
November 1, 2018November 1, 2018
#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach
October 30, 2018
FAP Not Common in Patients with Charcot-Marie-Tooth Disease Type 2, Study Reports
October 23, 2018October 23, 2018

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
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
September 20, 2018September 20, 2018

News

Rare Case of FAP and Mitochondrial Disease Reported in Study
September 18, 2018September 18, 2018
The rare case of a patient in Italy who had familial amyloid poyneuropathy (FAP) in addition ...
News

Somatostatin Analogues May Ease Chronic Diarrhea in FAP Patients, Pilot Study Reports
September 11, 2018September 11, 2018
Treatment with somatostatin analogues helps to ease the symptoms of difficult-to-treat diarrhea associated with ...
News

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 ...
News

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 ...
News

Blood Vessel Response to Vasodilator Agent Can Help Determine FAP Severity, Study Suggests
August 28, 2018August 28, 2018
A noninvasive evaluation of the response of skin blood vessels to a vasodilator compound ...
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 ...

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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