The More family was recently able to take a trip to Marin County, California to tour the Biomarin facility.
The company manufactures the medication (naglazyme) that Jasper More, 7, has been taking via weekly infusion for the past four and half years.
The medication is an artificial version of an important enzyme which is missing or lacking in Jasper’s body due to a condition know as MPS VI. There is a defective gene in his DNA that prevents the proper amount of enzyme to be created naturally.
MPS VI causes severely debilitating symptoms including: corneal clouding, bone and joint dysfunction, clawed hands, stunted growth and organ failure. By receiving the artificial copy of the enzyme, these symptoms are drastically slowed down so that life expectancy can be extended.
“We are very grateful … to all of the great people at Biomarin, first of all for what they do every day in making this product, but also for taking us in and making us feel welcome and gladly showing us their facility and process for making naglazyme,” stated Darren More.
“We were surprised to learn that it means as much for their employees to meet actual patients and families as it does for us to see and meet them. It was very humbling.”
While naglazyme slows the disease, it is still just a treatment. The More family continues to work diligently with The Isaac Foundation in search of a cure for MPS VI.
The More family thanked Equal Access For Rare Disorders for making all of the arrangements and making this “trip of a lifetime” possible.