- Remember all the children and adults who suffer from MPS and related diseases.
- Think about the children we have lost.
- Think about the doctors and scientists who are dedicated to finding a cure for MPS and related diseases.
- Appreciate each other and are thankful for the strength and support we both give and receive.
We honor Dr. Emil Kakkis, who developed Aldurazyme®, the enzyme replacement therapy for MPS I, and whose company, Ultragenyx, today announced a Phase 1/2 study of UX003 for MPS VII. Read the full press release. Read the Q&A with Dr. Emil Kakkis.