FDA Approves Rocket Pharma's Kresladi Gene Therapy for Ultra-Rare LAD-I After Setbacks on March 27, 2026 Approved gene therapy Kresladi Leukocyte adhesion deficiency type 1 Rocket Pharma ultra-rare disease United States Food and Drug Administration +
Gene Therapy Delivers Long-Term Immune Protection in Children With Rare Immune Disorders on October 16, 2025 Adenosine deaminase deficiency Combined immunodeficiency disease gene therapy immune protection Leukocyte adhesion deficiency type 1 Long-term Severe (severity modifier) +