Posts by: Marta Figueiredo PhD

Marta Figueiredo PhD

Marta Figueiredo holds a master’s in evolutionary and developmental biology and a PhD in biomedical sciences from the University of Lisbon, Portugal. Her research is focused on the role of several signaling pathways in thymus and parathyroid glands embryonic development.

April 1, 2021April 1, 2021

1st CRISPR Gene Editing Trial Slated to Open in Severe SCD Patients

News

The U.S. Food and Drug Administration approved the start of a first clinical trial of CRISPR_SCD001, the first non-viral and CRISPR/Cas9-based gene editing therapy for sickle cell disease (SCD). Both the ... Read more

February 19, 2021February 25, 2021

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

News

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare ... Read more

February 16, 2021February 16, 2021

Alternatives to Opioids Seen to Ease Pain, Raise Serotonin Levels in Early Study

News

A good diet and companionship, or treatment with an antidepressant called duloxetine was seen to significantly lessen pain in a mouse model of sickle cell disease (SCD), a study reports. Notably, ... Read more

February 9, 2021February 9, 2021

NHLBI Challenges Students to Create Innovative Tools to Raise Awareness

News

A new National Heart, Lung, and Blood Institute (NHLBI) challenge will award a first prize of $25,000 to a team of college or graduate students who develop an innovative tool ... Read more

January 14, 2021January 14, 2021

BCH-BB694 Gene Therapy Safely Treating Severe SCD Patients in Pilot Trial

News

BCH-BB694 — an experimental gene therapy targeting the BCL11A gene — safely increased the levels of fetal hemoglobin and prevented disease-associated complications in six people with severe sickle cell disease ... Read more

January 12, 2021January 12, 2021

Oral IMR-687 Seen to Lessen VOCs, Pain Crises in Patients in Phase 2a Trial

News

Six months of treatment with oral IMR-687 safely and effectively reduced the number of vaso-occlusive crises (VOCs) and other disease-associated pain crises in adults with sickle cell disease (SCD), updated data ... Read more

January 5, 2021January 5, 2021

CTX001 Continues to Prevent VOCs in People With Severe Disease, Trial Finding

News

CTX001, an experimental gene-editing cell therapy, safely and effectively increased the levels of fetal hemoglobin and prevented vaso-occlusive crises (VOCs) in three people with severe sickle cell disease (SCD), according ... Read more

December 8, 2020December 8, 2020

SCD Patients Benefit from Early Rivipansel Treatment for VOCs, New Analyses Show

News

Starting treatment with rivipansel (GMI-1070) shortly after vaso-occlusive crisis (VOC) onset significantly shortened hospital stays and the time to opioid discontinuation in children and adults with sickle cell disease (SCD), ... 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 a ... Read more