News

December 22, 2020December 22, 2020

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

News

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

News

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

News

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

News, syndicated

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

News

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

November 24, 2020November 24, 2020

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

News

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 ... Read more

November 20, 2020November 18, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

News, syndicated

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 ... Read more

November 17, 2020November 18, 2020

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

News

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 ... Read more

November 10, 2020November 10, 2020

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

News

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 ... Read more