A United States-based patient-reported adult polyglucosan body disease registry: initial results.

Jacy SparksFrancesco MichelassiJohn L P ThompsonRichard BuchsbaumNatacha PiresJanet T DeRosaKristin EngelstadSalvatore DiMauroHasan Orhan AkmanMichio Hirano

Published in: Therapeutic advances in rare disease (2024)

Although from a demographically constricted survey, the results provide basic clinical information for future studies to develop treatments for APBD.

Keyphrases

patient reported
current status
cross sectional
case control
health information
healthcare