News

Novartis’ investigational therapy for sickle cell disease (SCD), crizanlizumab (SEG101), was seen to reduce the number of patients reporting a pain crisis — including in those with a recent history of multiple crises — a post-hoc analysis of the SUSTAIN Phase 2 study reports. More patients (35.8%)…

A five-year partnership among The Sickle Cell Disease Association of America (SCDAA), the Sickle Cell Foundation of Georgia (SCFG), and the American Red Cross has been launched to encourage blood donation, with focus on African-Americans. As part of the partnership, educational campaigns to generate awareness about sickle cell disease…

A major distribution agreement with Cardinal Health will make Endari (L-glutamine) available in pharmacies nationwide that use Cardinal as their supplier, Emmaus Life Sciences announced. Already available to sickle cell disease patients in 36 states, Washington, D.C., and Puerto Rico, this agreement further expands the distribution network of…

Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights…

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…