News

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

November 5, 2020November 4, 2020

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

News, syndicated

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 ... Read more

November 3, 2020November 5, 2020

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

News

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 ... Read more

October 27, 2020October 27, 2020

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

News

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 ... Read more

October 26, 2020October 28, 2020

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

News, syndicated

A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar ... Read more

October 22, 2020October 23, 2020

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

News, syndicated

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a ... Read more

October 20, 2020October 20, 2020

UK Approves Phase 1 Trial of Potential Gene Editing Therapy, NTLA-2001

News

A Phase 1 trial of NTLA-2001, a potential gene editing therapy for familial amyloid polyneuropathy (FAP), was approved to open by the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA). Intellia ... Read more

October 13, 2020October 13, 2020

After Promising Early Data on Gene Editing, Intellia Will Launch ATTR Trial This Year

News

A single dose of NTLA-2001, Intellia Therapeutics’ investigational gene editing therapy for transthyretin amyloidosis (ATTR), results in a sustained reduction in the levels of the damaging transthyretin (TTR) protein ... Read more