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 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 ... 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 ... 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), ... 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 ... 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 12, 2020November 12, 2020

FT-4202, Oral SCD Therapy in Clinical Testing, Named Orphan Drug in EU

News

FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD) by Forma Therapeutics, has been designated an orphan drug by the European Medicines Agency (EMA). The decision was based on a positive ... Read more

October 15, 2020October 15, 2020

$7.1M NIH Grant Supports Study of Opioid Alternatives to Manage SCD Pain

News

The National Institutes of Health (NIH) has awarded a $7.1 million grant to University of Illinois Chicago (UIC) researchers to evaluate the effectiveness of two therapeutic strategies as alternatives to opioids in the ... Read more

October 8, 2020December 1, 2020

Rivipansel Granted FDA’s Rare Pediatric Disease Designation for SCD

News

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to GlycoMimetics’ investigational therapy rivipansel (GMI-1070) for the treatment of sickle cell disease (SCD). This designation is ... Read more

June 16, 2020July 9, 2020

Bluebird Bio Presents Data from LentiGlobin Gene Therapy Trial

News

Bluebird Bio’s investigational gene therapy LentiGlobin safely prevents serious vaso-occlusive crises and acute chest syndrome in adolescents and adults with severe sickle cell disease (SCD), according to recent data from ... Read more