Posts by: Marta Figueiredo

Marta Figueiredo

Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.

January 12, 2021January 12, 2021

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

News

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

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 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 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

September 15, 2020September 15, 2020

Actin Protein Alterations May Underlie TTR-induced Neurodegeneration

News

The characteristic toxic buildup of mutant transthyretin (TTR) protein clumps in familial amyloid polyneuropathy (FAP) affects the development of nerve cell fibers by interacting with regulators of a protein ... Read more

July 21, 2020July 21, 2020

Long-term Onpattro Use Safely Halts FAP Progression, Phase 2 Data Show

News

Long-term treatment with Onpattro (patisiran) safely and effectively halts or reverses disease progression in people with familial amyloid polyneuropathy (FAP), final data from a two-year Phase 2 trial show. ... Read more

November 29, 2018November 29, 2018

Researchers Report First Case of FAP with Chronic Dry Cough in Large Chinese Family

News

Researchers have reported the first case of familial amyloid polyneuropathy (FAP) with chronic dry cough — caused by a less common mutation — in a large Chinese family. The ... Read more

August 14, 2018August 14, 2018

FDA Approves Alnylam’s Onpattro (Patisiran) as FAP Therapy

News

The U.S. Food and Drug Administration (FDA) has approved Alnylam Pharmaceuticals’ Onpattro (patisiran) as the first-ever treatment for patients in the U.S. with familial amyloid polyneuropathy (FAP). “Today’s historic approval marks ... Read more

July 24, 2018July 24, 2018

Additional Data from APOLLO Trial Supports Patisiran as Promising FAP Therapy

News

Alnylam Pharmaceuticals’ investigative patisiran treatment improves overall health and prevents worsening of walking ability in patients with familial amyloid polyneuropathy (FAP), according to a new analysis from the APOLLO Phase ... Read more

May 8, 2018May 8, 2018

Rare Mutation Identified in Libyan Patient with Late-onset FAP, Case Study Reports

News

Researchers identified a very rare mutation in a Libyan man who was diagnosed with late-onset familial amyloid polyneuropathy (FAP), according to a case report. The report, titled “Familial amyloid ... Read more