Sickle Cell

Investigators find boys treated with alkylating agents or with hydroxyurea for sickle cell disease resulted in a significant reduction in spermatogonial cell counts, suggesting that these agents can affect fertility.

The New England Journal of Medicine has published the 48-week phase 3 clinical trial results that supported the FDA approval of L-glutamine oral powder for the reduction of acute complications of sickle cell disease in adult and pediatric patients.

New findings suggest that a specific hemoglobin production protein could be a target for therapy in sickle cell disease or beta-thalassemia.

FDA Commissioner, Scott Gottlieb, MD, announced the FDA’s efforts to advance the development of gene therapies through the addition of 6 scientific guidance documents.

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

Positive top-line data from Part A of a recent phase 3 trial evaluating voxelotor for the treatment of sickle cell disease have been released.

Updated 24-week data was released from patients treated with the 900 mg dose of voxelotor in the ongoing HOPE-KIDS 1 Study, a phase 2a open-label study in adolescents ages 6 to 17 years with sickle cell disease (SCD).

Sickle Cell Disease Association of America (SCDAA) and Emmaus Life Sciences partner in an effort to combat sickle cell disease.

In honor of World Sickle Cell Awareness Day, we’ve rounded up the big treatment advances made this year thus far for sickle cell disease.

The US Food and Drug Administration (FDA) has accepted the Bioverativ's Investigational New Drug (IND) application for BIVV003, a gene-edited cell therapy candidate for the treatment of people with sickle cell disease.

Altemia, a new treatment for sickle cell disease (SCD), was granted orphan drug designation.

The U.S. FDA has approved hydroxycarbamide/hydroxyurea tablets in pediatric patients suffering from sickle cell anemia, ages two years and older, France-based Addmedica has announced.

Imara has announced that it has dosed the first patient in its Phase 2a clinical trial of IMR-687 in adult patients with sickle cell disease.

Emmaus Life Sciences has announced that it has a Medicaid Drug Rebate Agreement with the Centers for Medicare and Medicaid Services, allowing coverage of Endari (L-glutamine oral powder).

Emmaus Life Sciences has announced that Endari, a medication developed to reduce acute complications in patients with sickle cell disease, is now available by prescription in the United States.

BioMarin Chief Executive Officer Jean-Jacques Bienaime spoke about the company’s development of valoctocogene roxaparvovec, a gene therapy in development for the treatment of Hemophilia A.

Global Blood Therapeutics, Inc. was granted Breakthrough Therapy Designation from the U.S. Food and Drug Administration for voxelotor for the treatment of sickle cell disease.

L-glutamine is the first SCD treatment approved for pediatric patients, and the first adult treatment approved in nearly 20 years.

When SCD patients' presentation accelerates, clinicians need to look further, and consider endocrine problems as possible causes.

Barbara Cappelli, MD, Eurocord International Registry, Paris, France, shared an overview from her team's study that found further evidence that for patients with severe sickle cell disease who have an HLA-identical sibling available as a donor, HSCT can be safe and effective.

Sickle cell disease, as debilitating as it is, is associated with the progressive complication of renal damage – beginning with hyerfiltration in childhood to glomerular filtration rate (GFR) decline and chronic renal failure in approximately 12 % of HbSS sickle cell adults.

Pain crises are common in those with sickle cell disease, but now new research may have identified biomarkers for patients whose pain becomes chronic.

Thirty-day emergency room readmissions due to acute pain is not uncommon in children with sickle cell disease, and researchers suspect that opioid prescription practices has something to do with it.

Although peripheral blood stem cell transplant (PBSCT) can cure sickle cell disease, the treatment runs the risk of permanent infertility.

A study published in the Journal of Clinical Sleep Medicine found 44 percent of adults with sickle cell disease also experience sleeping difficulties.

The medical marijuana program in Connecticut will expand to cover several additional conditions, including psoriatic arthritis.

Doctors may be undertreating patients for sickle cell disease, a US National Institutes of Health (NIH) panel said. A group of experts convened by the NIH's National Heart, Lung, and Blood Institute issued new guidelines Sept. 9, calling for more aggressive treatment of these patients including periodic blood transfusions for pediatric patients. They also called strongly for greater use of hydroxyurea, a drug that promotes production of one type of healthy hemoglobin and thus dilute the amount of the faulty hemoglobin that causes the symptoms of sickle cells disease.

Research has found that the prevalence of hyperuricemia and gout in adult patients with sickle cell disease in France was not a clinical problem..

Researchers have discovered that ion channel disorders may account for one-third of unexplainable sudden cardiac death in young people.