FDA Approves First Gene Therapy for Severe Leukocyte Adhesion Deficiency Type I
WHY IT MATTERS
Patients with severe LAD-I now have access to the first FDA-approved gene therapy for their condition, which could potentially offer a cure rather than just managing symptoms.
The FDA has approved a new treatment called Kresladi for a rare immune system disorder called Leukocyte Adhesion Deficiency Type I (LAD-I). This treatment works by using gene therapy, which means it fixes the faulty gene that causes the disease. This is the first gene therapy approved for this specific condition, giving patients with LAD-I a new treatment option.
The U.S. Food and Drug Administration today approved Kresladi (marnetegragene autotemcel), the first gene therapy for the treatment of severe Leukocyte Adhesion Deficiency Type I (LAD-I).
YOU CAN ACT ON THIS
If you or a family member has been diagnosed with severe LAD-I, contact your doctor immediately to discuss whether Kresladi is appropriate and how to access it.