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.

June 25, 2020June 25, 2020

Mitapivat Safely Increases Hemoglobin Levels in SCD Patients, Phase 1 Trial Shows

News

Mitapivat (AG-348) safely increased the amount of hemoglobin and changed the levels of biomarkers of red blood damage and energy production in adults with sickle cell disease (SCD), early ... Read more

June 23, 2020June 23, 2020

First SCD Patient Treated With CTX001 Remains VOCs-free After 9 Months, Trial Results Shows

News

The first patient with severe sickle cell disease (SCD) who received a single infusion of CTX001, an experimental gene-editing cell therapy, remains free of vaso-occlusive crises (VOCs) nine months ... Read more

June 4, 2020June 4, 2020

Sickle Cell Society Survey Looks to Better Understand Pandemic’s Affect

News

The Sickle Cell Society of the U.K. has created a survey to gather feedback from people with sickle cell disease (SCD) and their caregivers during the COVID-19 pandemic. Overall, the society aims ... Read more

June 2, 2020June 2, 2020

IMR-687 Safely Increases Fetal Hemoglobin Levels in SCD Patients, Phase 2a Trial Shows

News

IMR-687 safely increases the levels of fetal hemoglobin and the number of fetal hemoglobin-producing cells in people with sickle cell disease (SCD), according to early data from an ongoing ... Read more

May 14, 2020May 14, 2020

Agios to Decide Next Steps for Mitapivat as SCD Treatment by Mid-year

News

Agios Pharmaceuticals announced that, despite the current COVID-19 outbreak, it remains on track to decide by mid-year next steps for mitapivat (AG-348) as a potential treatment of sickle cell disease. Mitapivat is in an ongoing ... Read more

May 12, 2020May 12, 2020

Gene-editing Therapy CTX001 Granted RMAT Status by FDA for Severe SCD

News

The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to CTX001, an investigational gene-editing cell therapy, for the treatment of severe sickle cell disease ... Read more

May 7, 2020May 7, 2020

Sickle Cell Association Issues COVID-19 Guidelines for US Reopening

News

An advisory committee of the Sickle Cell Disease Association of America is recommending that people with sickle cell disease (SCD) be considered a “vulnerable” population during the reopening of the U.S. ... Read more

April 30, 2020April 30, 2020

APCER Report Confirms Safety of Sickle Cell Therapy Endari

News

APCER Life Sciences has released a report confirming the safety profile of Endari (L-glutamine), according to Emmaus Life Sciences, which developed the therapy. Endari is an oral therapy for ... Read more

April 28, 2020April 28, 2020

Fera Requests FDA Orphan Drug Designation for Naproxcinod for SCD

News

Fera Pharmaceuticals submitted an application to the U.S. Food and Drug Administration (FDA) requesting orphan drug status for naproxcinod, a potential treatment for for sickle cell disease (SCD). Orphan ... Read more

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