Updates

March 19, 2019March 19, 2019
Implanting Pacemaker Before Liver Transplant Does Not Lead to Better Outcomes, Study Reveals

Although most patients with familial amyloid polyneuropathy (FAP) who undergo liver transplant will require a pacemaker, implanting the device before the transplant is not ... Read more
March 15, 2019March 14, 2019
Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as ... Read more
March 12, 2019March 12, 2019
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases ... Read more
March 5, 2019March 5, 2019
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the ... Read more
February 26, 2019February 26, 2019
WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., ... Read more

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News

Vienna to Host RARE2019 Meeting on Rare Diseases
February 20, 2019February 20, 2019
About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city ...
News

FAP More Diverse Than Previously Believed, Review Study Contends
February 19, 2019February 19, 2019
A review of 542 cases of hereditary transthyretin amyloid polyneuropathy (ATTR-PN) from more than ...
News

Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
February 12, 2019February 12, 2019
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities ...
News

Eye Issues Still Prevalent in Val30Met FAP Patients After Liver Transplant, Study Shows
February 5, 2019February 5, 2019
Eye problems due to transthyretin (TTR) deposition are more prevalent among patients who have ...
News

FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases
February 1, 2019
The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug ...
News

Vyndaqel Is Safe, Effective for People with TTR-FAP, Review Study Suggests
January 29, 2019January 29, 2019
Treatment with Vyndaqel (tafamidis) is a safe, non-invasive way to delay neurologic progression of ...

Heart Problems Are Top Cause of Death for FAP Patients Who Had Liver Transplant, Study Says
January 22, 2019January 22, 2019
Cardiac problems are the leading cause of death among transthyretin familial amyloid polyneuropathy (ATTR, ...
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 ...
Vyndaqel May Help Halt Neurological Disease Progression in Patients With Val30Met Mutation, Study Reveals
January 8, 2019January 8, 2019
Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish
December 21, 2018December 21, 2018
FAP Patients with Val30Met Mutation Exhibit Differences in Japan, Study Finds
December 20, 2018December 20, 2018
Patients in France with FAP, Upper Limb Nerve Damage Often Misdiagnosed, Study Suggests
December 13, 2018December 13, 2018

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
Liver Transplant, Vyndaqel Prolong Life of Early-stage FAP Patients, Study Reports
November 15, 2018November 15, 2018
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
The distortion and subsequent atrophy of cells in the peripheral nervous system, due to ...
#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

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 ...
News

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 ...
News

#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 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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