FDA Clears First Gene Therapy For Rare Blood Disorder, Opening Door to New Treatment Class

The FDA's March 26, 2026 approval of Kresladi (marnetegragene autotemcel) for severe Leukocyte Adhesion Deficiency Type I marks a turning point not just for pediatric immunology, but for how rare disease marketers must operate. Rocket Pharmaceuticals now holds the first-ever FDA-approved gene therap

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This episode was published on April 27, 2026.

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