Beam Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to approve ristoglogene autogetemcel, or risto-cel, its single-dose, gene-edited cell therapy candidate for people with sickle cell disease (SCD), as early as year’s end. The application is expected to be supported by now-published interim data from…
Agios Pharmaceuticals is planning to file an application with the U.S. Food and Drug Administration (FDA) in the coming months that will seek accelerated approval of its oral therapy mitapivat for sickle cell disease (SCD). This decision by Agios follows a prefiling meeting with the regulatory agency in…
The Japan Agency for Medical Research and Development (AMED) — a national funding agency created to support medical R&D in the Asian nation — has awarded up to $32 million to Epifrontier Therapeutics to advance the clinical development of EPF-001, its investigational therapy for sickle cell disease (SCD)…
Discussion
As the season shifts from winter to spring, I’ve found myself reflecting deeply on what this past winter has meant for me. Historically, winter has been the hardest part of my journey with sickle cell disease, marked by crisis flare-ups, increased pain, and a constant awareness of…
Becoming educated and informed about sickle cell disease is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your sickle cell disease journey.
A stem cell transplant is the only potential cure for sickle cell disease, but there are a variety of other treatment approaches. Here you can find more information about current treatments as well as experimental therapies in the pipeline.
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