Mucopolysaccharidosis (MPS) is an inherited metabolic disease in which the body does not produce enough enzymes to break down sugars into simpler molecules or produces enzymes that do not work correctly. Over time, molecules called glycosaminoglycans (sugar carbohydrates in each of our cells that help build bone, cartilage, tendons, corneas, skin, and connective tissue) collect in the cells, blood, and connective tissues, resulting in permanent, progressive cellular damage.
In general, most affected people appear healthy at birth and experience a period of normal development, followed by a decline in physical and/or mental function. As the condition progresses, it may affect appearance; physical abilities; organ and system functioning; and, in most cases, cognitive development.1
Several distinct clinical types of Mucopolysaccharidosis have been identified.2
- MPS I, Hurler, Hurler-Scheie, Scheie
- MPS II, Hunter syndrome
- MPS III, Sanfilippo syndrome
- MPS IV, Morquio syndrome
- MPS VI, Maroteaux-Lamy syndrome
- MPS VII, Sly syndrome
- MPS IX, Natowicz disease
When choosing a specialty pharmacy to support Mucopolysaccharidosis (MPS) patients, consider a team experienced in rare diseases, ready to listen, provide counseling on treatment, help with side-effect management, and assist with sourcing third-party financial assistance. Read more about Annabelle’s journey with MPS and how Orsini has supported her and her family through the process. As part of your MPS patient’s treatment, Orsini Specialty Pharmacy provides the following:
Access to MPS Medications: Choose a specialty pharmacy with access to MPS medications. Orsini Specialty Pharmacy has access to Aldurazyme (laronidase) for MPS I, Elaprase (idursulfase) for MPS II, Vimizim (elosulfase alfa) for MPS IV and Naglazyme (galsulfase) for MPS VI.
MPS Care Team: Patients need an experienced MPS Care Team who can provide information and support throughout their journey. This includes a primary contact, clinical education, side-effects management, scheduled refill calls, and a direct phone, fax, and email address.
Shipping & Coordination of Care: Patients receive free priority overnight delivery of medications and necessary medical supplies. Orsini manages care coordination between prescribers, care sites, and patients.
Financial Assistance: MPS medications can be costly, and many patients cannot afford the co-pays associated with their health insurance. Orsini helps identify and assist in enrolling patients into manufacturer co-pay and foundation support programs.
24/7 Access to a Pharmacist: Choose a specialty pharmacy that can provide access to a pharmacist 24/7/365 to ensure patients always receive care when they need it.
We invite you to read more about Annabelle’s journey with MPS and how Orsini has supported her and her family through the process.
If you are a prescriber or physician office representative, click here to access MPS enrollment forms. If you are a prospective patient, click here to start your patient enrollment form. Also, you can always reach out to your Orsini rep to schedule a call and answer any questions.