University of California

‘Night and day’: Adventuring through childhood with newfound immune systems, thanks to newly FDA-approved therapy

After years of hospital stays, these kids are thriving thanks to a gene therapy that just earned accelerated FDA approval.

RCKT Stock Down Despite FDA Nod to Gene Therapy in Rare Disease

Rocket Pharmaceuticals drops despite the FDA's accelerated approval of gene therapy Kresladi in severe leukocyte adhesion ...

FDA approves gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder

Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role in a clinical trial has culminated in the first-ever U.S. Food and Drug ...
Pharmabiz

Rocket Pharma receives US FDA approval of Kresladi for paediatric patients with severe leukocyte adhesion deficiency-I

Rocket Pharmaceuticals, Inc., a fully integrated biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, announced that the US Food and ...
News-Medical.Net

FDA approves first gene therapy for rare pediatric immune disorder

The rare pediatric disease affects approximately one in one million children globally. Mutations in the ITGB2 gene disrupt ...
Yahoo Finance

Rocket Pharmaceuticals Announces FDA Approval of KRESLADI™ for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)

CRANBURY, N.J., March 27, 2026--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated biotechnology company advancing a sustainable pipeline of genetic therapies for rare ...
Yahoo

Rocket’s gene therapy Kresladi wins FDA approval in rare immune disease

Add Yahoo as a preferred source to see more of our stories on Google. The US Food and Drug Administration (FDA) has approved Rocket Pharmaceuticals’ Kresladi (marnetegragene autotemcel) in leukocyte ...
Seeking Alpha

Rocket Pharmaceuticals Announces FDA Approval of KRESLADI™ for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)

Severe LAD-I is an ultra-rare, life-threatening pediatric genetic immunodeficiency characterized by recurrent infections and high early-childhood mortality without treatment FDA grants Rare Pediatric ...