More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…
The blood level of a small protein called IP-10 could be a way to determine inflammation in pregnant women with sickle cell disease (SCD), according to new research in potential biomarkers. The exploratory study, “Chemokines in pregnant women with sickle cell disease,” appeared in the journal…
In a survey, patients across the U.K. with sickle cell disease — especially adolescents and young adults — voiced discontent with their healthcare experiences, particularly as they relate to emergency care and the promptness of pain relief, a study reports. The study, “Patient-reported experience measure in sickle cell disease,” was published in…
The month of September is filled with activities recognizing National Sickle Cell Awareness Month in the United States, all aimed at an ultimate cure for sickle cell disease (SCD). Clinical trials to accelerate development of new gene and cell-based therapies within the next decade have already begun as part of…
GlycA, a biomarker for conditions linked to vascular inflammation, has been found to not be a reliable indicator of inflammation in patients with sickle cell disease (SCD) due to its inability to differentiate between acute pain crisis and their usual state of health, according to researchers. The study, “GlycA…
Sancilio Pharmaceuticals‘ investigational oral compound Altemia (SC411) shows promising results in treating children with sickle cell disease (SCD), a Phase 2 clinical trial reveals. The study, “Double-blind, randomized, multicenter phase 2 study of SC411 in children with sickle cell disease (SCOT trial),” was published in…
McKesson Plasma and Biologics LLC will distribute Endari, a therapy indicated to reduce the acute complications of sickle cell disease (SCD) in adult and pediatric patients age 5 and older. The announcement was made by the Endari’s developer, Emmaus Life Sciences, Inc.. This distribution agreement enables…
Hoffmann-La Roche has sold the worldwide rights to its discontinued heart disease drug, inclacumab, to Global Blood Therapeutics (GBT) for $2 million and milestone payments. GBT plans to investigate inclacumab as a treatment for painful blockages of blood vessels in patients with sickle cell disease (SCD). The technology transfer process…
Siklos (hydroxyurea 100 mg tablets) — indicated for the treatment of pediatric patients, ages 2 and older, with sickle cell anemia (SCA) — can now be ordered in U.S. pharmacies. Developed by French pharmaceutical company ADDMEDICA, Siklos is now exclusively marketed in the U.S. by Medunik USA…
The National Institutes of Health (NIH) has awarded $4 million to the departments of emergency medicine and hematology at the Icahn School of Medicine at Mount Sinai Hospital in New York City to test a creative new approach for treating sickle cell disease (SCD). The strategy involves inhaled corticosteroids that…
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