MPS I treatment typically involves Hematopoietic Stem Cell Transplantation (HSCT), Enzyme Replacement Therapy (ERT), or a combination of both, along with supportive care such as pain management, anti-inflammatory medications, oxygen therapy, and surgery. While these MPS I treatments help manage symptoms and improve survival, they cannot reverse existing damage. Early intervention is essential to achieve the best possible outcomes.
ALDURAZYME (developed by BioMarin and Sanofi) is the only therapy approved by the US FDA for treating MPS I. ALDURAZYME was approved in the US and EU4 (France, Germany, Italy, and Spain) and the UK in 2003 and in Japan in 2006. It is indi...
