Posts by: Joana Carvalho

Joana Carvalho

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.

March 4, 2021March 4, 2021

VEST Lowers Duration of Painful SCD Crises

News

Vascular electrical stimulation therapy (VEST), a form of local electrical stimulation applied to the skin, can lower the duration of painful vaso-occlusive crises (VOCs) in patients with sickle cell disease ... Read more

March 2, 2021March 2, 2021

Stem Cell Transplant, Gene Therapy in SCD Tied to Improved Quality of Life

News

The quality of life of people with sickle cell disease (SCD) and transfusion-dependent thalassemia (TDT) — two inherited blood disorders — tends to improve if they receive a stem cell ... Read more

February 18, 2021February 18, 2021

Bluebird Bio Pauses Trials of Potential Gene Therapy LentiGlobin

News

Bluebird Bio has paused two clinical trials of LentiGlobin, its gene therapy candidate for sickle cell disease (SCD), after learning that a patient who received treatment more than five years ... Read more

February 11, 2021February 11, 2021

Oxbryta Can Ease or Fully Heal Leg Ulcers, Trial Analysis Shows

News

Oxbryta (voxelotor) can help ease, or fully heal, painful leg ulcers in people with sickle cell disease (SCD), according to a post-hoc analysis of a Phase 3 trial. These promising ... Read more

January 21, 2021January 21, 2021

FDA Clears Way for Trial of Gene Editing Therapy for Severe SCD

News

The U.S. Food and Drug Administration (FDA) has cleared the initiation of a Phase 1/2 trial investigating EDIT-301, Editas Medicine’s experimental gene editing cell therapy for sickle cell disease (SCD). The ... Read more

January 19, 2021January 26, 2021

Clinical Trial Cleared for GPH101, First Potentially Curative SCD Therapy

News

The investigational gene editing therapy GPH101 will be the first potentially curative treatment for sickle cell disease (SCD) to be tested in a Phase 1/2 clinical trial. The U.S. Food ... Read more

December 15, 2020December 15, 2020

Emmaus Support Program Gives US Patients Low-cost Access to Endari

News

Emmaus Life Sciences has launched a program that will allow eligible sickle cell disease patients in the U.S. to access its Endari (L-glutamine) medication at low cost or free of ... Read more

December 3, 2020December 3, 2020

GBT Launches Early Access Program for Voxelotor in Europe, Other Regions

News

Global Blood Therapeutics (GBT) has launched an early access program for voxelotor in Europe and other regions outside the U.S. to allow people with hemolytic anemia associated with sickle cell ... Read more

November 3, 2020November 3, 2020

EU Approves Adakveo to Treat Pain Crises in SCD Patients, 16 and Older

News

The European Commission has approved Novartis’ Adakveo (crizanlizumab) as a preventive treatment for recurrent vaso-occlusive crises (VOCs) in people, 16 and older, with sickle cell disease (SCD). The approval covers ... Read more

October 20, 2020October 20, 2020

Cyclerion Suspends Development of Olinciguat for Sickle Cell Disease

News

Cyclerion Therapeutics has decided to suspend the development of olinciguat, its investigational oral therapy for sickle cell disease (SCD), after the treatment failed to show adequate activity in a Phase ... Read more