The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how…
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Children and adolescents with sickle cell anemia do less physical activity and have a more sedentary lifestyle than healthy controls, according to a study. The study, “Objectively measured physical activity levels and sedentary time in children and adolescents with sickle cell anemia,” was published in PLOS…
The Sickle Cell Foundation of Georgia (SCFG) will host its 42nd Annual Camp New Hope July 14-20 for youngsters living with sickle cell disease (SCD). The fun and enrichment camp will be held a week earlier than usual at one of Georgia’s most historic campsites, Rock…
Acute vaso-occlusive pain is associated with the onset of menstruation, and about 25% of the women with sickle cell disease can differentiate these episodes from menstrual-cramp pain, a study found. Findings in the study, “Acute Vaso-Occlusive Pain is Temporally…
Sickle cell disease (SCD) patients were safely treated with a phosphodiesterase 9A (PDE9) inhibitor therapy called PF-04447943 with no serious side effects reported and possible evidence of fewer vaso-occlusion pain crises, a Phase 1b clinical trial reports. Safety and early signs of possible efficacy — including a drop in biomarkers linked…
Howard University President Wayne A.I. Frederick has unveiled a campaign to raise awareness and funds for the school’s Center for Sickle Cell Disease. “I am proud to officially launch the Run to Cure Sickle Cell campaign,” said Frederick, MD, MBA, FACS, in a press release. “At birth, I was…
Randomized controlled trials are needed to examine the benefits of vaccinating people with sickle cell disease (SCD) against salmonella infections, which can be particularly fatal to children with the disease, a study says. The review study, “Vaccines for preventing invasive salmonella infections in …
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Crizanlizumab Designated FDA Breakthrough Therapy for Potential in Vaso-occlusive Crisis Prevention
Crizanlizumab (SEG101), Novartis‘ investigational compound for sickle cell disease (SCD), received breakthrough therapy designation from the U.S. Food and Drug Administration for its potential to prevent vaso-occlusive crises (VOCs). The FDA designation was granted based on promising data from the company’s multicenter, randomized, double-blind, 52-week, Phase…
The U.S. Food and Drug Administration (FDA) has awarded fast track status to investigational therapy CTX001 to treat sickle cell disease. A Phase 1/2 clinical trial (NCT03745287) is recruiting patients. CTX001 uses the CRISPR gene-editing technology to increase the production of fetal hemoglobin in patients’ red blood cells. Fetal hemoglobin is…
The U.S. opioid epidemic is not linked to higher in-hospital mortality in patients with sickle cell disease (SCD), according to a national study. The study came about due to the pronounced increase in opioid-related overdoses and deaths in the U.S. that has been reported since 2000. Its researchers did…
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