On May 15th, Project Alive posted the first episode of the ongoing documentary series to recognize International MPS Awareness Day.
Each following episode will provide the audience with a glimpse into the lives of families dealing with Hunter syndrome (mucopolysaccharide II or MPS II). The goal of the series is to raise awareness of Hunter syndrome, as well as the need for a gene therapy for the lysosomal storage disorder.
In Hunter syndrome, patients are missing the iduronate sulfatase enzyme, resulting in an accumulation of dermatan and heparan sulphate. Common symptoms may include stunted growth, coarse facial features, hearing loss, joint stiffness, sleep apnea, thickening of heart valves, obstructive airways, enlarged liver and spleen, and cognitive impairment.
There is currently an approved enzyme replacement therapy for the condition [Elaprase (idursulfase)], but it is widely believed that gene therapy is necessary so that the persons can make their own iduronate sulfatase throughout the body.
Enter Project Alive, whose initiative is to fund until a gene therapy gets into clinical studies.
The trailer of the series is below, and those interested can visit projectalive.org to see the first episode. Future episodes will be uploaded every 2-3 weeks.