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Mission Statement

The National MPS Society exists to find cures for MPS and related diseases. We provide hope and support for affected individuals and their families through research, advocacy and awareness of these devastating diseases.

Since 1974 the Society has supported individuals and families affected with MPS and related diseases. We are governed by a member-elected volunteer Board of Directors, many of whom are parents of children with MPS. We benefit from the expertise of a Scientific Advisory Board, comprised of world-class physicians, researchers and medical professionals throughout the world.

MPS and Related Diseases

Mucopolysaccharidoses (MPS) and related diseases are genetic lysosomal storage diseases (LSD) caused by the body's inability to produce specific enzymes. Normally, the body uses enzymes to break down and recycle materials in cells. In individuals with MPS and related diseases, the missing or insufficient enzyme prevents the proper recycling process, resulting in the storage of materials in virtually every cell of the body. As a result, cells do not perform properly and may cause progressive damage throughout the body, including the heart, bones, joints, respiratory system and central nervous system. While the disease may not be apparent at birth, signs and symptoms develop with age as more cells become damaged by the accumulation of cell materials.

What are the Major Characteristics of MPS and Related Diseases?

While the symptoms of the diseases may vary from one syndrome to another, there are many similarities. Affected individuals often have mental retardation, cloudy corneas, short stature, stiff joints, incontinence, speech and hearing impairment, chronic runny nose, hernia, heart disease, hyperactivity, depression, pain and a dramatically shortened life span.

Research is helping today's families

Although there is currently no cure for MPS, research is making great strides. Carrier detection, the development of replacement enzymes, and the possibility of gene therapy are among today's research themes and treatment options. Bone marrow and cord blood transplantation have been considered successful for many, though relatively few individuals qualify for this high-risk procedure. We've made major advancements in research thanks to the fundraising efforts of the Society and its members.



About Us | MPS & Related Diseases | Treatments & Clinical Trials | News & Events | Membership | Family Support
Library | Research Grants | Pharmaceutical Partners | Legislative | Fundraisers | Walk/Run
Donate | Press Room | Tributes | Home | Gallery | MPS Logo Shop

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