Posts by: Joana Carvalho, PhD

Joana Carvalho, PhD

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.

February 4, 2020February 4, 2020

SCD Patients Receiving Hydroxyurea in the U.S. Still Face Many Challenges

News

People with sickle cell disease (SCD) treated with hydroxyurea in the U.S. still face many challenges, including the financial burden posed by their healthcare expenses, low treatment adherence and ... Read more

January 30, 2020January 30, 2020

FDA-Approved Therapies for Sickle Cell Disease May Not be Cost-Effective, ICER Draft Report Says

News

FDA-approved therapies for sickle cell disease (SCD) may not be cost-effective at their current prices, according to a draft report issued by the Institute for Clinical and Economic Review ... Read more

January 23, 2020January 23, 2020

FDA Names Potential Gene Therapy for Sickle Cell an ‘Orphan Drug,’ Aruvant Announces

News

A potential gene therapy for sickle cell disease (SCD) and beta-thalassemia, known as ARU-1801, has been named an orphan drug by the U.S. Food and Drug Administration (FDA), a status ... Read more

January 21, 2020January 21, 2020

FT-4202, FORMA’s Experimental SCD Therapy, Fares Well in Phase 1 Trial of Healthy Volunteers

News

FT-4202, FORMA Therapeutics’ experimental disease-modifying therapy for sickle cell disease (SCD), shows a favorable safety and pharmacokinetic profile in healthy volunteers, according to data from a Phase 1 clinical ... Read more

January 9, 2020January 9, 2020

FDA Grants Rare Pediatric Disease Status to Investigational Gene Therapy ARU-1801 for Sickle Cell

News

The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to ARU-1801, Aruvant’s experimental gene therapy for the treatment of sickle cell disease (SCD) and beta-thalassemia. This ... Read more

January 7, 2020January 22, 2020

GBT and Syros Partner to Develop Sickle Cell Treatments That Boost Fetal Hemoglobin

News

Global Blood Therapeutics (GBT) and Syros Pharmaceuticals have teamed up to discover, develop, and market new therapies for sickle cell disease (SCD) and beta thalassemia. Under the collaboration agreement, ... Read more

January 2, 2020January 3, 2020

NIH Grant Backs Project to Develop New Pain Therapy for SCD Patients

News

The National Institutes of Health (NIH) has awarded a grant to researchers at Carnegie Mellon University’s College of Engineering to support their project to develop a non-invasive, non-toxic and non-addictive pain ... Read more

December 12, 2019December 12, 2019

Gene Therapy LentiGlobin Continues to Show Promising Results, Phase 1/2 Trial Data Shows

News

LentiGlobin, Bluebird Bio’s investigational gene therapy for sickle cell disease (SCD), continues to show promising results in SCD patients participating in the company’s Phase 1/2 HGB-206 clinical trial, according ... Read more

December 5, 2019December 5, 2019

Adakveo Being Tested in Series of Trials, Including at Higher Dose and in Children

News

A Phase 3 trial is testing a higher dose of Adakveo (crizanlizumab), the first approved targeted treatment for sickle cell disease, to explore if that dose might be more ... Read more