August 17, 2017
AbbVie’s Imbruvica is First Approved Treatment for Adults with Chronic Graft-Versus-Host-Disease

The U.S. Food and Drug Administration (FDA) approved Imbruvica (ibrutinib) for treatment of adult patients with chronic graft-versus-host-disease (cGVHD) who failed ... Read more
August 3, 2017
Sancilio Completes Enrollment in Clinical Trial of Altemia as Treatment for Children with Sickle Cell Conditions

Sancilio Pharmaceuticals has completed enrolling patients in a Phase 2 clinical trial evaluating Altemia (docosahexaenoic acid) as a treatment for children with ... Read more
August 1, 2017August 1, 2017
Gamida Cell Receives $3.5M Grant from Israeli Government to Help Develop CordIn Therapy

The Israeli government has awarded Gamida Cell a $3.5 million grant to advance the development of clinical trials for CordIn, a therapy ... Read more
July 27, 2017July 27, 2017
‘Natural’ Gene Mutation May Offer Way of Treating Sickle Cell Disease, Study Says

Introducing a “natural” genetic mutation into the blood cells of people with sickle cell and like diseases, using CRISPR gene editing, was seen to restore ... Read more
July 25, 2017
Sema4 and Helix Launch CarrierCheck, a Genetic Screen of 67 Hereditary Diseases

Sema4, an interdisciplinary health information company, has partnered with Helix, a personal genomics company, to launch CarrierCheck — a simple, ... Read more
July 20, 2017
Quest Diagnostics Launches Genetic Test to Assess Risk for Sickle Cell Anemia, Other Inherited Disorders

Couples who wish to know the risk of passing on a genetic disease such as sickle cell anemia to ... Read more
July 19, 2017July 19, 2017
Localized Nerve Anesthesia a Viable Option for Pain Control in Pediatric SCD Patients, Study Suggests

Doctors treating pediatric patients with sickle cell disease (SCD) for pain associated with a vaso-occlusive crisis (VOC) often have ... Read more
July 11, 2017
Global Blood Expands Sickle Cell Study to Include Kids Ages 6-11

Global Blood Therapeutics has expanded its ongoing HOPE-KIDS 1 trial (NCT02850406), evaluating the investigative GBT440 in pediatric patients with sickle ... Read more
July 7, 2017July 7, 2017
FDA Approves Endari, 1st Sickle Cell Treatment in Almost 20 Years, for Patients Age 5 and Older

Endari (L-glutamine) today became the first therapy in almost 20 years to win approval from the U.S. Food and ... Read more
July 6, 2017
Researchers Identify Genetic Predictors of Sickle Cell Anemia-Related Complications

Patients with sickle cell anemia who do not have alpha-thalassemia and the genetic rs1427407 T variant of the BCL11A gene may ... Read more
June 29, 2017
European Agency Grants PRIME Access to GBT440 Therapy for Treatment of SCD

The European Medicines Agency (EMA) has granted GBT440 — Global Blood Therapeutics’ lead drug candidate for sickle cell disease (SCD) — access ... Read more
June 27, 2017June 27, 2017
Chip-based Models Mimic Organs to Advance Understanding of Sickle Cell Disease

Researchers at Mississippi State University (MSU) are using tiny polymer chips to build models to help them better understand sickle ... Read more

AbbVie’s Imbruvica is First Approved Treatment for Adults with Chronic Graft-Versus-Host-Disease

The U.S. Food and Drug Administration (FDA) approved Imbruvica (ibrutinib) for treatment of adult patients with chronic graft-versus-host-disease (cGVHD) who failed to respond to other forms of systemic therapy, AbbVie announced. FDA’s ... Read more

August 3, 2017

Sancilio Completes Enrollment in Clinical Trial of Altemia as Treatment for Children with Sickle Cell Conditions

Sancilio Pharmaceuticals has completed enrolling patients in a Phase 2 clinical trial evaluating Altemia (docosahexaenoic acid) as a treatment for children with sickle cell anemia (SCA) and sickle cell disease (SCD). The ... Read more

August 1, 2017August 1, 2017

Gamida Cell Receives $3.5M Grant from Israeli Government to Help Develop CordIn Therapy

The Israeli government has awarded Gamida Cell a $3.5 million grant to advance the development of clinical trials for CordIn, a therapy to treat sickle cell disease (SCD) and thalassemia. The money, granted by the ... Read more

July 27, 2017July 27, 2017

‘Natural’ Gene Mutation May Offer Way of Treating Sickle Cell Disease, Study Says

Introducing a “natural” genetic mutation into the blood cells of people with sickle cell and like diseases, using CRISPR gene editing, was seen to restore the production of fetal hemoglobin and offer a way to treat these ... Read more

July 25, 2017

Sema4 and Helix Launch CarrierCheck, a Genetic Screen of 67 Hereditary Diseases

Sema4, an interdisciplinary health information company, has partnered with Helix, a personal genomics company, to launch CarrierCheck — a simple, saliva-based test that screens for 67 conditions, including sickle cell ... Read more

July 20, 2017

Quest Diagnostics Launches Genetic Test to Assess Risk for Sickle Cell Anemia, Other Inherited Disorders

Couples who wish to know the risk of passing on a genetic disease such as sickle cell anemia to their children may now do so with QHerit, a new ... Read more

July 19, 2017July 19, 2017

Localized Nerve Anesthesia a Viable Option for Pain Control in Pediatric SCD Patients, Study Suggests

Doctors treating pediatric patients with sickle cell disease (SCD) for pain associated with a vaso-occlusive crisis (VOC) often have few optimal therapy options. But a recent study reviewed a ... Read more

July 11, 2017

Global Blood Expands Sickle Cell Study to Include Kids Ages 6-11

Global Blood Therapeutics has expanded its ongoing HOPE-KIDS 1 trial (NCT02850406), evaluating the investigative GBT440 in pediatric patients with sickle cell disease, to include a new single-dose cohort in younger ... Read more

July 7, 2017July 7, 2017

FDA Approves Endari, 1st Sickle Cell Treatment in Almost 20 Years, for Patients Age 5 and Older

Endari (L-glutamine) today became the first therapy in almost 20 years to win approval from the U.S. Food and Drug Administration to treat sickle cell disease. In giving the green ... Read more

July 6, 2017

Researchers Identify Genetic Predictors of Sickle Cell Anemia-Related Complications

Patients with sickle cell anemia who do not have alpha-thalassemia and the genetic rs1427407 T variant of the BCL11A gene may be at higher risk for hemolysis (rupture of red blood ... Read more

June 29, 2017

European Agency Grants PRIME Access to GBT440 Therapy for Treatment of SCD

The European Medicines Agency (EMA) has granted GBT440 — Global Blood Therapeutics’ lead drug candidate for sickle cell disease (SCD) — access to its Priority Medicines (PRIME) program. GBT440 is an oral, ... Read more

June 27, 2017June 27, 2017

Chip-based Models Mimic Organs to Advance Understanding of Sickle Cell Disease

Researchers at Mississippi State University (MSU) are using tiny polymer chips to build models to help them better understand sickle cell disease. Research on the models, which can also be ... Read more