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Gene Therapy Shows Lasting Benefit for Children With Rare Disorder

F or children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational ...

Rocket Pharmaceuticals Announces FDA Acceptance of BLA Resubmission of KRESLADI™ for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable ...

Experimental gene therapy successfully revives immune systems in children with deadly disorder

A groundbreaking gene therapy has successfully restored immune function in children with ADA-SCID, a severe genetic disorder.