News

January 21, 2020January 21, 2020

Eye Disease Common in FAP Patients with Ser77Tyr Mutation, May Serve as Biomarker, Research Suggests

News

People with a type of FAP caused by a Ser77Tyr mutation are more likely to develop conjunctival lymphangiectasia, an uncommon clinical condition characterized by swelling of the conjunctiva — ... Read more

January 14, 2020January 15, 2020

PTC Announces Tegsedi’s Launch Into Brazilian Market as Treatment for FAP

News

Tegsedi (inotersen) has been approved in Brazil to treat stage 1 or 2 nerve damage (polyneuropathy) in adults with hereditary transthyretin amyloidosis (hATTR), also known as familial amyloid polyneuropathy (FAP), ... Read more

January 7, 2020January 7, 2020

FAP Annual Costs and Burden Are Considerable, But in Line with Other Rare Diseases

News

The annual costs and disease burden associated with hereditary transthyretin amyloidosis polyneuropathy (ATTRv-PN), also known as familial amyloid polyneuropathy (FAP), are considerable but within the range of other rare ... Read more

December 17, 2019December 17, 2019

Early Diagnosis of Eye Manifestations Essential in FAP Patients, Review Says

News

Although uncommon, eye manifestations can affect patients with familial amyloid polyneuropathy (FAP). These are mostly caused by opacities in the jelly liquid of the eye and usually treated by ... Read more

December 10, 2019December 10, 2019

Diuretics to Treat Blood Pressure May Stop Proteins from Clumping, Early Study Suggests

News

Two FDA-approved treatments for high blood pressure, diuretics called indapamide and hydrochlorothiazide, might also treat diseases associated with amyloid deposits, such as familial amyloid polyneuropathy (FAP), a study suggests. ... Read more

December 6, 2019December 6, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

News

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s ... Read more

December 3, 2019December 3, 2019

Alnylam Opens ‘Advocacy for Impact’ Grant Program Supporting FAP, Like Patient Groups

News

Alnylam Pharmaceuticals has opened its second annual competitive grants program, supporting patient advocacy projects worldwide that aim to address the unmet and diverse needs of ATTR amyloidosis, a group of ... Read more

November 26, 2019November 26, 2019

Akcea and Ionis Launch Phase 3 Trial of AKCEA-TTR-LRx in People With FAP

News

Akcea Therapeutics and Ionis Pharmaceuticals announced the launch of a Phase 3 clinical trial evaluating the safety and efficacy of AKCEA-TTR-LRx (ION-682884) for the treatment of adults with familial ... Read more

November 19, 2019November 19, 2019

Gait Tracking Using RGB-D Camera Could Aid Doctors in Diagnosing, Managing FAP, Study Shows

News

An affordable, portable, and non-invasive human motion tracking system based on an RGB-D camera can be useful for monitoring gait impairments in people with familial amyloid polyneuropathy (FAP), a ... Read more

November 15, 2019November 16, 2019

Rare Disease Film Festival Highlights Patient and Researcher Unity

News

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. ... Read more

Pin It on Pinterest