Fly a paper airplane to help raise awareness about mucopolysaccharidosis (MPS)!
Help Connexion Healthcare in our fight against rare disease by joining with us on May 15 for MPS Awareness Day. The National MPS Society is sponsoring events nationwide, encouraging people everywhere to engage in social media activities (go here to see the social media calendar) designed to raise awareness about MPS and support patients and their families.
What is mucopolysaccharidosis?
Mucopolysaccharidoses are a group of rare lysosomal storage disorders. Lysosomes are structures inside cells that contain enzymes for breaking down and recycling debris inside almost all of the cells of the body; without this breakdown of debris, materials accumulate inside the cell, with devastating effects on the body.
MPS is a rare disease
MPS is a rare, inherited disorder that occurs when a child receives a recessive gene for the disease from each parent. MPS is seen in only 1 out of 25,000 to 50,000 births. MPS has 7 different subtypes, including Hurler syndrome, one of the most severe forms of the disease. The subtypes are classified depending on which specific lysosomal enzyme is missing, and may have a range of severity within a single subtype. As medical communication experts in the rare disease space, we understand that disease awareness is integral to quicker diagnoses and innovative new therapies.
What does MPS do to the body?
MPS affects the cardiovascular, skeletal, respiratory, and central nervous systems. The disease usually strikes at a young age, with symptoms appearing as the cells continually accumulate an excess of cellular materials. Children typically develop significant impairments, such as mental retardation, short stature, incontinence, speech and hearing impairments, heart disease, hyperactivity, depression, pain, and a substantially shortened life span.
Can MPS be treated?
Certain types of MPS can be treated by replacement of the missing enzyme; 4 of the 7 MPS subtypes have FDA-approved enzyme replacement therapies. Although no currently available therapy cures the disease, treatment can lessen the symptoms. Bone marrow transplantation may also be somewhat effective under certain circumstances, but it is risky and few patients qualify as candidates for the procedure. Genetic testing and preconception counseling can help prospective parents learn whether they possess recessive genes for MPS and about the risks of passing on the gene, so that they can make informed decisions about family planning. One of the many goals of MPS Awareness day is to help draw attention to treatment options.
Connexion Healthcare’s Rare Disease Center of Excellence works with industry and the community
Connexion Healthcare is the first healthcare communication agency to specialize in the therapeutic area of rare disease, and as such, we are committed to partnering with both industry and patient advocacy groups to increase awareness of rare diseases and orphan drugs. Our Rare Disease Center of Excellence has great expertise in communicating the science behind rare diseases and orphan drugs. We strategically partner with industry leaders in the rare disease community to proactively connect all stakeholders in launching products to underserved patient populations. Our team consists of skilled communications experts who plan, develop, and disseminate scientific communications based on their advanced knowledge of the science, the patient journey, healthcare providers, and the orphan drug approval process.
At Connexion Healthcare, our cross-functional teams navigate complicated treatment and market landscapes by elucidating the appropriate pathways to brand success. We understand how brand strategy drives market share. Since launching its dedicated Rare Disease Center of Excellence, Connexion Healthcare has developed prelaunch, launch, and postlaunch communications in numerous rare diseases and disorders: congenital and genetic, movement, metabolic, and lysosomal storage disorders (such as MPS); lung, blood, skin, and endocrine diseases; and rare cancers.
For further information regarding the Rare Disease Center of Excellence at Connexion Healthcare and how we can develop rare disease communications to differentiate therapies by their unique attributes, contact:
Susan Stein, MPH
SVP, Strategic Services and Business Development