Nature

Prompt recovery of recipient hematopoiesis after two consecutive haploidentical peripheral blood SCTs in a child with leukocyte adhesion defect III syndrome

Leukocyte adhesion deficiency (LAD) type III is a rare syndrome characterized by severe recurrent infections, leukocytosis and increased bleeding tendency. All integrins are normally expressed; yet, a ...
Nature

Leukocyte Adhesion Deficiency and Stem Cell Transplantation

Leukocyte adhesion deficiency (LAD) represents a group of rare, genetically determined immunodeficiency disorders that impair the ability of white blood cells to adhere to vascular endothelium, ...
Business Wire

Rocket Pharmaceuticals Presents Positive Top-line Data from Severe Leukocyte Adhesion Deficiency-I Program at the 25th Annual Meeting of the American Society of Gene and Cell ...

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies ...
Nasdaq

Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy Designation for RP-L201 Gene Therapy for Leukocyte Adhesion Deficiency-I

— Enrollment Complete in Phase 1/2 Clinical Trial of RP-L201 for the Treatment of LAD-I — “Receiving RMAT designation and completing Phase 1/2 patient enrollment are important steps in advancing our ...
Business Wire

Rocket Pharmaceuticals Announces Clearance of IND for RP-L201 Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I)

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (Nasdaq:RCKT) (“Rocket”), a leading U.S.-based multi-platform gene therapy company, today announces the clearance of the Company’s ...