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Have you or your child recently been diagnosed with MPS or ML, or is your child undergoing diagnostic testing? We are here to help and can connect you with the information you need.
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Donations to the National MPS Society allow us to support families affected by MPS and ML, fund medical research to find cures for these diseases and keep the public engaged and informed.
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Support those affected by MPS and ML

Families are the linchpin of our community, and whether it’s financial assistance during a hard time, a special treat for an affected family member or to help make educational dreams come true, we support MPS and ML families across the nation.
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News

FDA approves MPS VII drug

The U.S. Food and Drug Administration today approved Mepsevii (vestronidase alfa-vjbk) to treat pediatric and adult patients with MPS VII, Sly syndrome. The safety and efficacy of Mepsevii, developed by Ultragenyx, were established in clinical trial and were tested on 23 patients ranging from 5 months to 25 years of age. Patients received treatment with … Continued

Sangamo treats first patient with gene therapy

Sangamo Therapeutics treated its first patient in the Phase 1/2 clinical trial (“the CHAMPIONS study“) evaluating SB-913, an investigational in vivo genome editing therapy for people with MPS II, Hunter syndrome. Sangamo aims to treat MPS II by using genome editing to insert a corrective gene into a precise location in the DNA of liver … Continued

Society awards MPS and ML research grants

The National MPS Society’s annual research program has awarded $405,000 in funding for additional research grants. Dr. Sharon Byers – $90,000 general grant over two years SA Pathology (WHC site) Adelaide, Australia “Can the cell cycle be re-set to normal in the MPS growth plate chondrocytes?” Dr. Igor Nestrasil – $60,000 MPS I grant Department … Continued

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