News

More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…

The California Institute for Regenerative Medicine (CIRM) awarded Matthew Porteus, a Stanford University associate professor of pediatrics, a $5.2 million grant to conduct the groundwork leading to a clinical trial of a possible treatment for sickle cell disease (SCD). SCD develops when a gene is mutated that codes for a…

As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…

The Medical University of South Carolina (MUSC) was recently awarded a Doris Duke Charitable Foundation grant to develop a new gene-modifying therapy for the treatment of sickle cell disease. The project, “Epigenetic Modulators for the Treatment of Sickle Cell Disease,” was awarded more than $750,000 as part of the…

The U.S. Food and Drug Administration (FDA) has awarded Dr. Kenneth Ataga, a $2 million grant over five years to support a prospective study in sickle cell anemia to determine biomarkers of specific cellular function changes in chronic kidney disease (CKD). Ataga, a researcher with the University of…

Researchers have found that patients with sickle cell disease (SCD) have increased risk of developing leukemia, but lower risk for breast cancer or male genital cancers. These findings resulted from a population-based study titled “Increased risk of leukemia among sickle cell disease patients in California” that was published in…

Sancilio Pharmaceuticals has completed a Phase 2 clinical trial evaluating Altemia (docosahexaenoic acid) as a treatment for children aged 5 to 17 with sickle cell disease (SCD). The company expects to release the main results of the SCOT trial (NCT02973360) by the end of 2017. The study assessed…

A “National Poll of African-Americans on Sickle Cell Disease Awareness” conducted earlier this year has revealed that, while most African-Americans are familiar with sickle cell disease (SCD), only about one-third are aware that it affects people of African descent in a disproportionate way. The findings highlight the need for more…

A Phase 1 clinical trial of Imara’s new investigational drug, IMR-687, showed that it was safe and well-tolerated in healthy volunteers, paving the way for a Phase 2 trial in adults with sickle cell disease (SCD). Imara, based in Cambridge, Massachusetts, plans to start a Phase 2 trial of IMR-687…

The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to Altemia Soft Gelatin Capsules, developed for the treatment of sickle cell disease (SCD) in children. Altemia is being developed by Sancilio Pharmaceuticals as an oral, once-daily formulation for children aged 5 to 17 years who…