City to pioneer child medicines

A new five year research programme based at Alder Hey Children’s Hospital has been set up to develop medicines and study the effectiveness of those used by children. (more) 

BioMarin Provides Update on Phenylase Product Development

BioMarin Pharmaceutical Inc. has announced that studies of Phenylase(TM) appear to have been successful in its development for the treatment of phenylketonuria (PKU). BioMarin plans is planning more studies of Phenylase and int+ends to file for an Investigational New Drug application (IND) with the U.S. Food and Drug Administration (FDA).(more) 

Orphan Europe and Peptimmune Partner to Develop Specific Immunotherapy for Life-Threatening Skin Disease

Orphan Europe sarl and Peptimmune Inc. have signed a Scientific Collaboration and License Agreement regarding the development of a peptide immunotherapy for the treatment of pemphigus vulgaris. The aim of the collaboration is to bring to market a new efficient drug to treat this rare, life-threatening, autoimmune skin disease.(more) 

Junctional epidermolysis bullosa - A first patient successfully treated by gene therapy

A team of Italian researchers has succeeded in treating a patient with the genetic skin disease junctional epidermolysis bullosa through the use of gene therapy. The successful trial could lead to further studies in France.(more) 

Studies Published in PNAS on the Mechanism of Amicus’ Experimental Treatment for Gaucher Disease

Amicus Therapeutics has announced that studies of the mechanism of action of its investigational treatment for Gaucher disease, AT2101 were published in the September 12 edition of the Proceedings of the National Academy of Sciences of the United States of America (PNAS). Amicus has filed an investigational new drug application for AT2101 for the treatment of Gaucher disease and Phase I clinical trials are underway.(more) 

New genetic link to cot death identified  

 A new research study shows that B=babies born with specific variants of three key genes are 14 times more likely to die from cot death. The findings build on earlier research by The University of Manchester team that had already associated one of these genes with the condition. The discover of further genes is a major step forward in understanding the causes of cot death or `sudden infant death syndrome' (SIDS).(more) 

UK scientists grow artificial liver

A tiny artificial liver has been grown from stem cells by Newcastle University scientists and is set to revolutionise the medical world. The breakthrough was made using stem cells taken from umbilical cords.