Sickle Cell Disease News

July 9, 2020July 9, 2020

Bronchodilators to Treat Asthma May Worsen Youngsters’ SCD, Study Suggests

Bronchodilators used to treat asthma could worsen sickle cell disease (SCD) in children and teens, and contribute to a greater risk of vaso-occlusive crisis (VOC), a study suggests. The study, ... Read more

July 7, 2020July 7, 2020

Potential Oral DMT Showing Safety and Signs of Efficacy in Phase 1 Trial

Sickle Cell Disease News

FT-4202 — an investigational treatment for sickle cell disease (SCD) — has a favorable safety profile and the expected biological effects, providing preliminary support of efficacy, according to data from an ... Read more

July 2, 2020July 6, 2020

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

Sickle Cell Disease 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 sickle ... Read more

June 30, 2020June 30, 2020

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

Sickle Cell Disease 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 with ... Read more

June 25, 2020June 26, 2020

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

Sickle Cell Disease 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 Phase ... Read more

June 23, 2020June 23, 2020

First SCD Patient Treated With CTX001 Remains VOCs-free After 9 Months

COVID-19 News, Sickle Cell Disease 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 after ... Read more

June 18, 2020June 18, 2020

World Sickle Cell Day Celebrates Patients, Raises Awareness

COVID-19 News, Sickle Cell Disease 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. Across ... Read more

June 16, 2020June 16, 2020

Bluebird Bio Presents Data from LentiGlobin Gene Therapy Trial

Sickle Cell Disease 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 a ... Read more

June 11, 2020June 11, 2020

GBT to Request That Oxbryta Treat Children Starting at Age 4

Sickle Cell Disease 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 announced ... Read more

June 4, 2020June 4, 2020

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

COVID-19 News, Sickle Cell Disease 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 to ... Read more

June 2, 2020June 2, 2020

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

Sickle Cell Disease 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 Phase ... Read more

May 29, 2020May 30, 2020

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

COVID-19 News, Sickle Cell Disease News

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies ... Read more

May 28, 2020May 28, 2020

Smartphone App May Help Assess Anemia Using Eyelid Pictures

Sickle Cell Disease News

A smartphone app that measures hemoglobin levels using pictures of a person’s inner eyelid may help assess anemia in people with blood disorders such as sickle cell disease, a new ... Read more

May 26, 2020May 26, 2020

Vitamin D Supplements May Reduce Pain-related Emergency Room Visits in Children with SCD

Sickle Cell Disease News

Taking vitamin D supplements may lower the number of visits to the emergency department due to vaso-occlusive pain episodes in African American children with sickle cell disease (SCD), a study found. The findings also ... Read more

May 21, 2020May 21, 2020

Partnership to Ensure Supply of LentiGlobin, Potential Gene Therapy

Sickle Cell Disease News

Bluebird Bio extended a partnership with two divisions of Hitachi Chemical for the long-term development and manufacturing of LentiGlobin, its investigational gene therapy for sickle cell disease (SCD), and other potential and approved treatments. The two ... Read more