February 13, 2017

We are very happy to announce that we have been awarded a RARE Impact Innovation Grant from Global Genes to conduct focus group sessions at our 2017 Family and Scientific Conference in a pilot study that will form the foundation for research studies addressing the unme...

January 12, 2017

Among medical professionals, rare diseases like Peroxisome Biogenesis Disorder-Zellweger Spectrum Disorder (PBD-ZSD) are challenging to recognize in patients and even more challenging to treat. Currently, there is no specific treatment for PBD-ZSD and most therapeutic...

January 10, 2017

Our dedicated researchers worked hard over the holidays, making great progress on the PBD-ZSD retinal gene therapy project at McGill University. The PEX1 mice have received retinal injections of the normal PEX1 gene. The mice are doing well and soon we will be able to...