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This microdocumentary shares the moving story of a family with two young sons who have severe hemophilia, giving us a glimpse into their history and treatment routine. Hemophilia is a rare bleeding disorder in which the body is unable to properly create a blood clot. Beyond external bleeding, like a cut, people with hemophilia also bleed internally and in their joints, which can lead to crippling arthritis later in life.
The story highlights the significant progress made in this therapeutic area – from the mother’s perspective as a child growing up with a brother who had hemophilia, to caring for her two young children today – and shares a message of excitement and hope for the future. Throughout the video, leading Cheladv74 researcher Debra Pittman explains what the future could bring in this therapeutic area, specifically in research areas like gene therapy.