FAP News Today

June 20, 2022June 20, 2022

9 Cases of Onpattro Used to Ease FAP Symptoms Described in Report

FAP News Today

Several cases in which treatment with Onpattro (patisiran) was used to ease familial amyloid polyneuropathy (FAP) symptoms were described in a recent report from Italy. The study, “Italian Real-Life Experience ... Read more

June 16, 2022June 17, 2022

Vutrisiran Approved by FDA to Treat FAP Under Brand Name Amvuttra

FAP News Today

Vutrisiran — now known by the brand name Amvuttra — has been approved by the U.S. Food and Drug Administration (FDA) to treat familial amyloid polyneuropathy (FAP), also known as ... Read more

June 13, 2022June 13, 2022

NORD’s ‘Living Rare, Living Stronger’ Patient, Family Forum Set for June 26

COVID-19 News, FAP News Today

The National Organization for Rare Disorders’ (NORD) “Living Rare, Living Stronger Patient and Family Forum” is back in person on June 26 for a day of learning and networking in ... Read more

June 6, 2022June 7, 2022

Vutrisiran Continues to Show Promise to Lessen Heart Damage

FAP News Today

Vutrisiran continues to show promise at lessening heart damage in adults with familial amyloid polyneuropathy (FAP) with cardiac involvement, according to exploratory new data from the ongoing Phase 3 HELIOS-A trial. Data ... Read more

May 23, 2022May 23, 2022

Fat Buildup in Large Muscle of Lower Leg May Signal FAP Progression

FAP News Today

Fat accumulation in the soleus muscle, a large muscle of the lower leg, may be indicative of disease progression in people with hereditary transthyretin (ATTRv) amyloidosis, a group of conditions ... Read more

May 16, 2022May 16, 2022

Genetic Screening for Mutations May Help in Early Diagnosis, Treatment

FAP News Today

A seven-year genetic screening program in Bulgaria involving people at high risk of hereditary transthyretin amyloidosis — a group of conditions that also includes familial amyloid polyneuropathy (FAP) — found patients ... Read more

May 9, 2022May 9, 2022

2nd Part Launches in Phase 1 Trial of Gene-editing Therapy NTLA-2001

FAP News Today

The first patient has been dosed in the second part of a Phase 1 trial evaluating NTLA-2001, Intellia Therapeutics’ investigational gene-editing therapy for people with familial amyloid polyneuropathy (FAP) and ... Read more

May 5, 2022May 5, 2022

Organizations Rally to Help Ukrainian Rare Disease Patients

FAP News Today

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ... Read more

May 2, 2022May 2, 2022

TTR Gene Variant Linked to Heart Failure in Black People in US

FAP News Today

A genetic variant in TTR — the gene implicated in familial amyloid polyneuropathy (FAP) — was linked with a greater risk of heart failure and death among Black people living ... Read more

April 25, 2022April 25, 2022

Europe Awaits Proposed New Framework for Sharing Health Data

FAP News Today

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health ... Read more

April 18, 2022April 18, 2022

Real-world Study: Tegsedi Slows FAP Progression, Preserves Life Quality

FAP News Today

In a real-world study of patients with worse disease severity at the start of treatment, Tegsedi (inotersen) slowed the progression of hereditary transthyretin amyloidosis — a group of disorders that ... Read more

April 13, 2022April 13, 2022

Telehealth Added to Latest Digital-friendly NORD State Report Card

COVID-19 News, FAP News Today

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues ... Read more

April 11, 2022April 11, 2022

FDA Pushes Decision on Vutrisiran to July After Packaging Site Issues

FAP News Today

The U.S. Food and Drug Administration (FDA) is extending by three months its review timeline for vutrisiran, an experimental therapy for familial amyloid polyneuropathy (FAP) developed by Alnylam Pharmaceuticals. A decision, ... Read more

April 4, 2022April 4, 2022

Small Vesicles in Blood Contribute to Toxic TTR Protein Buildup in FAP

FAP News Today

Small vesicles in the blood contribute to the clumping or aggregation, and toxic accumulation of the transthyretin (TTR) protein in people with familial amyloid polyneuropathy (FAP), a study shows. These ... Read more

March 28, 2022March 28, 2022

New Compound Score May Help Screen for TTR Genetic Testing

FAP News Today

A highly sensitive tool has been developed to help identify patients with familial amyloid polyneuropathy (FAP). Using a compound score based on clinical symptoms and electrophysiological data, clinicians can now ... Read more