Sickle Cell Disease News

June 17, 2021June 17, 2021

Oxbryta in HOPE-KIDS Meeting ‘Urgent Need’ of Children, Ages 4-11

In children with sickle cell disease (SCD), ages 4–11, weight-based doses of Oxbryta (voxelotor) result in similar rises in hemoglobin levels and reductions in red blood cell destruction (hemolysis) to ... Read more

June 16, 2021June 16, 2021

Get Ready for NORD’s Patient and Family Forum, Set for June 26-27

COVID-19 News, Sickle Cell Disease News

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the ... Read more

June 15, 2021June 15, 2021

Oxbryta Given Promising Innovative Medicine Status in UK

Sickle Cell Disease News

A Promising Innovative Medicine (PIM) designation has been granted to Oxbryta (voxelotor), a treatment to reduce the destruction of red blood cells in people, 12 and older, with sickle cell ... Read more

June 10, 2021June 10, 2021

Rare 2030 Action Campaign Pushing for European Plan for Rare Diseases

Sickle Cell Disease News

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in ... Read more

June 8, 2021June 8, 2021

Supporters Urged to Wear Red for World Sickle Cell Day on June 19

Sickle Cell Disease News

From sporting red gear to entering a spoken word contest, supporters globally are poised to mark World Sickle Cell Awareness Day, an annual event that takes place on June 19. ... Read more

June 3, 2021June 3, 2021

Advocates Can Try for Grants Ahead of Rare Disease Week on Capitol Hill

Sickle Cell Disease News

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in ... Read more

June 3, 2021June 3, 2021

Machine Learning Can Predict Rapid Kidney Function Decline

Sickle Cell Disease News

Machine learning tools can identify sickle cell disease (SCD) patients at high risk of progressive kidney disease as early as six months in advance, a study shows. The study, “Using ... Read more

June 1, 2021June 1, 2021

Endari Sickle Cell Therapy Now Under Review in Saudi Arabia

Sickle Cell Disease News

The Saudi Food and Drug Authority (SFDA), in Saudi Arabia, will review Emmaus Life Sciences’ application for the approval of its oral medication Endari (L-glutamine) for treating sickle cell disease (SCD). ... Read more

May 25, 2021May 25, 2021

Imaging Technique Predicts Risk of Vision Problems

Sickle Cell Disease News

Rapid and repeated imaging of blood flow through the eyes can inform physicians of a patient’s risk of sustaining eye damage and developing vision problems related to sickle cell disease ... Read more

May 20, 2021May 20, 2021

Gene Therapy ARU-1801 Showing Early Efficacy, Safety in Phase 1/2 Trial

Sickle Cell Disease News

ARU-1801, an experimental gene therapy for sickle cell disease (SCD), lowered the number of vaso-occlusive crises and led to blood cells producing fetal hemoglobin in a first group of three treated ... Read more

May 18, 2021May 18, 2021

US Nonprofits Team Up to Bolster Advocacy Efforts

Sickle Cell Disease News

Two U.S. nonprofits are partnering to tackle legislative issues that benefit sickle cell disease (SCD) patients and their families. The partnership between the Sickle Cell Disease Association of America (SCDAA) ... Read more

May 13, 2021May 13, 2021

Oxbryta Safe and Effective in Children Ages 4 to 11, Early Trial Data Indicates

Sickle Cell Disease News

Six months of treatment with Oxbryta (voxelotor) safely and effectively increased hemoglobin levels and reduced red blood cell destruction, or hemolysis, in children ages 4–11 with sickle cell disease (SCD), ... Read more

May 11, 2021May 11, 2021

Non-viral Gene Editing of Marrow Stem Cells Shows Potential in Early Study

Sickle Cell Disease News

A non-viral, CRISPR-based gene-editing tool showed a potential to reverse mutations that cause sickle cell disease (SCD) by successfully editing genes in stem cells within the bone marrow of mice. ... Read more

May 6, 2021May 6, 2021

Spoken Word Poetry Contest Open to Sickle Cell Patients, Caregivers

Sickle Cell Disease News

A spoken word poetry contest is welcoming submissions from people with sickle cell disease (SCD) and their caregivers. The contest, “Lift Every Voice to Shine the Light on Sickle Cell,” ... Read more

May 4, 2021May 4, 2021

FDA Expands Approvals of Ferriprox

Sickle Cell Disease News

The U.S. Food and Drug Administration (FDA) has expanded the label of Chiesi Global Rare Diseases’ iron-binding oral treatment Ferriprox (deferiprone) to include patients ages 3 and older who have ... Read more