Gene Therapy Promise to Fight “The Royal Disease”

Americans living with hemophilia (mostly men, as the genetic condition affects the X chromosome) have to live with care, prioritizing their safety and ability to pay for costly medical support. A single fall or bump can lead to a life-threatening bleed and medical bills can surpass $1 million a year.

New experimental gene therapies seem to offer hope for a cure but haven’t been in trials long enough to confirm lasting success. Still, “the general trajectory has been forward” for gene therapy and, for the 20,000 Americans living with hemophilia, this means hope for a life less burdened by the condition.

The New York Times

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