As part of a multi-year RWE initiative, AMCP Research Institute partnered with IQVIA, payers, pharmaceutical companies, and others to design payer-focused RWE standards that were published in JMCP in September, 2025.
JMCP Findings: Improving Patient Care in Mucopolysaccharidoses
The mucopolysaccharidoses (MPSs) are a group of rare disorders that cause widespread damage over time and lead to early death. They are hard to diagnose, so starting treatment can take a long time. Some MPS disorders do not have treatments, but even with treatment, many patients and families struggle. Researchers are studying new treatments but face problems because the disorders are rare. Health care professionals should know about these difficulties to improve patient care in MPS. This article provides key insights and suggested best practices for managed care to improve patient care in MPS disorders, which may also be relevant to other rare and ultra-rare conditions.
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Learn more about the AMCP Metabolic Health Initiative.
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