Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy.

This analysis aims to describe the outcomes of two nonambulatory patients with Duchenne muscular dystrophy (DMD) who participated in two clinical studies. The two consecutive trials of eteplirsen (studies 201 and 202) were conducted in patients with DMD (N = 12) and confirmed genetic mutations amenable to exon 51 skipping.In study 201, 12 patients were randomized to receive once-weekly, double-blind intravenous infusions of eteplirsen 30 or 50 mg/kg or placebo for 24 weeks; patients then received open-label eteplirsen during weeks 25 through 28.

The successful treatment of severe aplastic anemia with autologous cord blood transplantation.

Cord blood transplantation has been used extensively in the allogeneic setting for acquired and genetic disorders of hematopoiesis. There is less experience in the utility of autologous cord blood transplantation, and there is great controversy about the role of autologous cord blood collection and storage. We report on the successful use of autologous cord blood transplantation for the treatment of severe aplastic anemia following fulminant hepatic failure and living related liver transplantation.

New York State guidelines for cord blood banking. Department of Health.

In response to the profound interest in the transplantation of umbilical cord blood, the New York State (NYS) Department of Health embarked on a regulatory strategy designed to provide guidance to the practitioner while allowing for the flexibility required by the inherent developmental nature of the technology. The result was the development of the Guidelines for Collection, Processing, and Storage of Cord Blood Stem Cells, adherence to which is optional. It is anticipated that through the use of such guidelines as a supplement to the existing hematopoietic progenitor cell (HPC) regulations for tissue banks, practitioners may provide to potential patients the safest and most efficacious cord blood products currently available, and information gained through the use of these products will be of optimal value toward the goal of continuous improvement in the delivery of this new technology.

Cord blood stem cells: how to get them and what to do with them.

This article reviews the means of obtaining cells from the available reservoirs of cord blood, intended as sources of immature hematopoietic stem cells that ultimately could be useful for transplantation, gene therapy, and research. Various issues must be considered when collecting umbilical cord blood regardless of the method employed. One must regard the basic fetal-placental physiology and hemodynamic characteristics prior to and at the time of procurement.