News

January 19, 2021January 19, 2021

Imaging Agent Can Help Differentiate Between Amyloidosis Types

News

AT-01, an imaging agent being developed by Attralus, can help to differentiate between transthyretin (ATTR) amyloidosis — which includes familial amyloid polyneuropathy (FAP) — and other forms of amyloidosis, ... Read more

January 12, 2021January 12, 2021

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

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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). ... Read more

January 8, 2021January 8, 2021

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

News, syndicated

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD ... Read more

January 5, 2021January 5, 2021

Gout Medicine BBM May Be Potential FAP Treatment

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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 ... Read more

December 22, 2020December 22, 2020

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

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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 ... Read more

December 15, 2020December 15, 2020

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

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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 ... Read more

December 8, 2020December 8, 2020

Abdominal Fat Pad Biopsy Accurate at Diagnosing ATTR, Study Finds

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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 ... Read more

December 2, 2020December 1, 2020

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

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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 ... Read more

December 1, 2020December 1, 2020

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

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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 ... Read more

November 26, 2020November 25, 2020

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

News, syndicated

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 ... Read more