The recent signing into law by President Barack Obama of the 21st Century Cures Act, a bipartisan bill whose aims include speeding the development of treatments for patients with difficult medical conditions, like sickle cell disease (SCD), was welcomed by the biotech company Imara in a recent press release. The legislation, supported…
Scientists have created a risk profile that uses three genetic markers to determine whether there is a high or low chance of a sickle-cell-anemia (SCA) patient developing kidney disease. Preliminary results indicate that the combination does a better job of predicting kidney disease than markers used by themselves. The study,…
Results from the Phase 2 SUSTAIN clinical trial demonstrated that crizanlizumab (SEG101, Selexys Pharmaceuticals, Novartis), an anti-P-selectin antibody, reduced by 45.3% the median annual rate of sickle cell-related pain crises (SCPC), compared to placebo in patients with or without hydroxyurea therapy, a drug used to reduce the rate…
Imara recently presented positive preclinical results of its lead drug candidate IMR-687, a potent PDE9 inhibitor for the treatment of sickle cell disease (SCD). The presentation, which was previewed in a November article, took place at the 2016 American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego,…
The UT Physicians Comprehensive Sickle Cell Center has expanded its facilities in response to the needs of an increasing number of people living with sickle cell disease (SCD) who suffer from chronic pain, the university announced in a press release. The new center will offer sub-specialty, social…
Karolinska Development’s Modus Therapeutics has been issued two U.S. patents covering its proprietary drug sevuparin, a candidate for the treatment of sickle cell disease. Specifically, the two patents have been granted claims for the substance sevuparin and a manufacturing method for producing the drug (U.S. Patent 9480701) and for…
Imara is scheduled to present preclinical efficacy and safety data on IMR-687, its lead product candidate for the treatment of sickle cell disease (SCD), at the American Society of Hematology (ASH) 58th Annual Meeting and Exposition in San Diego, Calif., Dec. 3-6. The oral presentation, titled “A Novel,…
The U.S. Food and Drug Administration (FDA) has accepted for review a New Drug Application (NDA) from Emmaus Life Sciences for its orally administered pharmaceutical grade L-glutamine (PGLG) treatment for sickle cell disease. If the FDA approves this new treatment, it would be the first FDA-approved medicine available…
The U.S. Food and Drug Administration (FDA) has approved the use of Immucor’s PreciseType HEA test to screen blood donors for sickle cell trait (SCT), an inherited blood disorder that affects 1 million to 3 million Americans. SCT is not a disease, but people with the disorder…
Treatment outcomes for people with sickle cell disease (SCD) have improved significantly in the past few decades. Today, over 90 percent of children who are born in Western countries with the genetic condition will live to adulthood. This is a remarkable achievement, as SCD was once synonymous with childhood mortality…
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