RETRACTED: An Innovative Tool for Evidence-Based, Personalized Treatment Trials in Mucopolysaccharidosis

• Main Authors: Anna-Maria Wiesinger (Author), Brian Bigger (Author), Roberto Giugliani (Author), Christina Lampe (Author), Maurizio Scarpa (Author), Tobias Moser (Author), Christoph Kampmann (Author), Georg Zimmermann (Author), Florian B. Lagler (Author)
• Format: Book
• Published: MDPI AG, 2023-05-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Mucopolysaccharidosis (MPS) is a group of rare metabolic diseases associated with reduced life expectancy and a substantial unmet medical need. Immunomodulatory drugs could be a relevant treatment approach for MPS patients, although they are not licensed for this population. Therefore, we aim to pro...