News

July 2, 2020July 2, 2020

7th Annual Walk With the Stars & Move-A-Thon Kicks Off July 11 to Fight SCD

News

While it will be entirely virtual this year, the 7th Annual Walk With the Stars & Move-A-Thon is taking place July 11 to raise awareness and money to fight ... Read more

June 30, 2020June 30, 2020

GBT to Request Oxbryta’s Approval in Europe for SCD Patients 12 and Older

News

Global Blood Therapeutics (GBT) plans to submit a marketing authorization application to the European Medicines Agency (EMA) asking that Oxbryta (voxelotor) be approved for treating hemolytic anemia in people ... Read more

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 22, 2020June 22, 2020

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

cystic fibrosis, News, syndicated

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will ... Read more

June 18, 2020June 19, 2020

World Sickle Cell Day Celebrates Patients, Raises Awareness

News

Supporters are poised to mark World Sickle Cell Day, observed each June 19 to raise awareness about the red blood cell disorder, and to raise funds to fight it. ... Read more

June 16, 2020June 16, 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

June 11, 2020June 11, 2020

GBT to Request That Oxbryta Treat Children Starting at Age 4

News

Global Blood Therapeutics (GBT) will ask the U.S. Food and Drug Administration (FDA) to allow children with sickle cell disease (SCD), ages 4 to 11, to also be treated with Oxbryta (voxelotor). The company ... Read more

June 9, 2020June 10, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

News

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis ... 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