Clinical trials are research studies that determine whether treatments or devices are safe for humans. These studies also look for effective medical approaches for MPS and ML and help provide reliable data for patients, researchers and doctors. Clinical trials are conducted on small groups to determine whether a drug or procedure causes negative reactions or unsatisfactory side effects.
As clinical trial updates happen frequently, please check clinicaltrials.gov for up to date information on clinical trials, observational and natural history studies.
For MPS I, II, IVA, VI, and VII, a clinical trial through the University of California, San Francisco is available to those receiving a prenatal diagnosis to administer intrauterine enzyme replacement therapy. Please click here for additional information and contact firstname.lastname@example.org with any questions.