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…
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…
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…
For this year’s Solutions to Empower Patients (STEP) program, global healthcare company Novartis has announced it will fund up to five original and creative ideas to help sickle cell disease (SCD) patients and their families. Each selected proposal will be awarded up to $50,000. Specifically, Novartis is seeking…
Healthcare professionals, scientists and field experts learned about the latest advances in the diagnosis and treatment of sickle cell and related disorders at the 12th Annual Sickle Cell Disease and Thalassaemia (ASCAT) conference. Biopharmaceutical company Emmaus Life Sciences sponsored the Oct. 22-24 conference in London. Its theme was “Sickle…
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…
With drug overdoses now the leading cause of accidental death in the U.S., efforts are underway in many states to limit abuse and access to prescription opioids — steps that, some say, could cause unnecessary suffering among sickle cell disease (SCD) patients who rely on these medications to treat their symptoms. “I’m seeing…
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