A 16-year-old boy with sickle cell disease (SCD) was diagnosed with autosomal dominant polycystic kidney disease (ADPKD), an inherited condition that causes fluid-filled cysts to form in the kidneys, a case study reports The report, “Dual diagnosis of autosomal dominant polycystic kidney disease and sickle cell disease in…
The Sickle Cell Disease Association of America (SCDAA), in collaboration with Forma Therapeutics, is launching a campaign to raise awareness and boost knowledge about sickle cell disease (SCD). The campaign, called “Believe it!”, will be promoted on television and radio channels, encouraging people to go through the website…
The first patient has been dosed in a Phase 1/2 trial assessing the safety and preliminary efficacy of Graphite Bio’s experimental gene editing therapy GPH101 (now called nula-cel) in people with sickle cell disease (SCD). The trial, called CEDAR (NCT04819841), is currently recruiting participants at three sites,…
Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…
Several clinical research sites seeking to find new treatments for people with sickle cell disease (SCD) are joining a collaborative network created by the American Society of Hematology Research Collaborative (ASH RC). The initiative, called ASH RC Sickle Cell Disease Clinical Trials Network, has already engaged about 24,000 patients…
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