FAP News Today

January 12, 2021January 12, 2021

Vutrisiran Reduces Neurologic Damage, Improves Physical Function, Trial Data Show

FAP News Today

Vutrisiran, a second-generation RNA interference (RNAi) therapy candidate, safely and effectively reduces neurologic impairment and improves physical function and quality of life in adults with familial amyloid polyneuropathy (FAP). That’s ... Read more

January 5, 2021January 5, 2021

Gout Medicine BBM May Be Potential FAP Treatment

FAP News Today

Benzbromarone (BBM), a medicine used to treat gout, can stabilize the TTR protein to the same degree as therapies used to treat people with familial amyloid polyneuropathy (FAP), a study ... Read more

December 22, 2020December 22, 2020

TTR Amyloidosis in Afro-Caribbean Woman Underpins Disease’s Diversity

FAP News Today

The case of a woman of Afro-Caribbean descent with early onset transthyretin (TTR) amyloidosis, who was found to carry a TTR mutation more commonly seen in those of Celtic descent, ... Read more

December 15, 2020December 15, 2020

PRX004 Reduces Neuropathy Progression, Heart Strain in Familial ATTR, Trial Finds

COVID-19 News, FAP News Today

PRX004, an investigational treatment by Prothena, can reduce the progression of nerve damage (neuropathy) and improve heart function in people with hereditary transthyretin amyloidosis (ATTR), which includes familial amyloid polyneuropathy (FAP), ... Read more

December 8, 2020December 8, 2020

Abdominal Fat Pad Biopsy Accurate at Diagnosing ATTR, Study Finds

FAP News Today

Abdominal fat pad biopsy is an accurate diagnostic tool for patients with suspected hereditary transthyretin amyloidosis (ATTR), showing the greatest sensitivity in those with ATTR with polyneuropathy, also known as familial ... Read more

December 2, 2020January 3, 2021

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

FAP News Today

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) ... Read more

December 1, 2020December 1, 2020

Onpattro Shows Sustained Benefits, Safety in FAP Patients in 1-year Extension Study

FAP News Today

Onpattro (patisiran) results in sustained improvements in polyneuropathy, quality of life, and other symptoms among people with familial amyloid polyneuropathy (FAP), while showing an acceptable safety profile, according to a one-year ... Read more

November 26, 2020December 28, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

COVID-19 News, FAP News Today

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a ... Read more

November 24, 2020November 24, 2020

Onpattro Better Than Tegsedi in Treating FAP, Indirect Trial Comparison Says

FAP News Today

Onpattro (patisiran) is more effective than Tegsedi (inotersen) at reducing neurological impairment and improving quality of life in people with familial amyloid polyneuropathy (FAP), a comparison of data from two clinical ... Read more

November 20, 2020December 24, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

FAP News Today

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how ... Read more

November 17, 2020November 19, 2020

3 FAP Therapies Win 2020 Prix Galien USA Awards for Best Biotechnology Product

FAP News Today

Three recently approved therapies for treating familial amyloid polyneuropathy (FAP), also known as hereditary transthyretin amyloidosis with polyneuropathy, have won their developers Best Biotechnology Product honors in the 2020 Prix ... Read more

November 10, 2020November 10, 2020

First ATTR Patient Dosed in Phase 1 Trial of Intellia’s Gene-editing Therapy NTLA-2001

FAP News Today

The first patient has been dosed in a Phase 1 clinical trial evaluating NTLA-2001, Intellia Therapeutics’ investigational gene-editing therapy for the treatment of hereditary transthyretin amyloidosis with polyneuropathy (ATTR-PN), also known ... Read more

November 5, 2020December 7, 2020

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

FAP News Today

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations ... Read more

November 3, 2020November 3, 2020

Phase 3 Trial of Acoramidis in ATTR-CM Patients Fully Enrolled, Trial in FAP Underway

FAP News Today

Eidos Therapeutics announced that the Phase 3 clinical trial evaluating its lead candidate acoramidis (formerly known as AG10) in treating adults with transthyretin amyloidosis cardiomyopathy (ATTR-CM) is fully enrolled. Top-line results from part A ... Read more

October 27, 2020October 27, 2020

TTR Variant, E61K, That’s Not Prone to Clumping Seen to Cause FAP in Other Ways

FAP News Today

Although less able to form damaging protein deposits, the E61K variant of transthyretin appears to cause familial amyloid polyneuropathy (FAP) through other mechanisms, a study suggested. The study, “A low ... Read more