Factor IX (FIX) exhibits significant extravascular distribution and binds type IV collagen in the subendothelial extracellular matrix in the extravascular space, which influences its function and phar...

Researchers report first cancer caused by virus widely used to deliver genes, but say risk is low

Fidanacogene elaparvovec is a single-dose gene therapy designed to express the high-activity factor IX (FIX) variant FIX-R338L. Participants (N = 15) …

The novel treatment, partly developed by CHOP, eliminates the need for regular infusions while preventing the life-threatening bleeds caused by the condition.

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The order applies to the entirety of new and, possibly, ongoing research grant dollars that go to universities and academic medical centers.

The associate director of clinical in vivo gene therapy at Children’s Hospital of Philadelphia discussed a case he presented at the American Society of Gene and Cell Therapy’s 2025 meeting.

Effort to fund new gene therapy study spotlights rare disease with elusive remedy

People with Williams Syndrome treat strangers as their new best friends. Now the condition is revealing clues to our evolutionary past – and what makes us human.

/PRNewswire/ -- Researchers at Children's Hospital of Philadelphia (CHOP) in collaboration with researchers at Royal Prince Alfred Hospital in Sydney,...

Ryan White changed perceptions of the disease in the United States.

In a recent interview, the health secretary also suggested that the measles vaccine had harmed children in West Texas, center of an outbreak.

A child who was not vaccinated has died from measles in West Texas. It's the first death in an outbreak that began late last month and the first from measles in the U.S. since 2015.

Blood Podcast | Blood | American Society of Hematology Blood Podcast   The editors of Blood are pleased to introduce a podcast series, published weekly in conjunction with each iss...

The Spurs are expected to be without superstar big man Victor Wembanyama for the rest of the season due to a blood clot in his shoulder.

Select a state on the map to see the impact of NIH funding across America.