Mucopolysaccharidoses | National Institute of Neurological …
Mucopolysaccharidoses (MPS) are a group of rare inherited metabolic diseases. There are many different types of MPS which present a variety of physical and neurological symptoms. Glycosaminoglycans, also known as mucopolysaccharides, are long chains of sugars (carbohydrates) in our cells.
National MPS Society - We Help Cure, Support, & Advocate for …
Our mission is to serve those impacted by mucopolysaccharidosis (MPS) and mucolipidosis (ML). These are rare genetic diseases with no cures. We support research, provide families with invaluable resources, and work to increase public and …
Epidemiology of Mucopolysaccharidoses Update - PMC
In this study, we aimed to update the prevalence of MPS across the world. We have collected and investigated 189 publications related to the prevalence of MPS via PubMed as of December 2020. In total, data from 33 countries and 23 regions were compiled and analyzed.
Ultragenyx Puts BLA for MPSIII Gene Therapy UX111 in Front of FDA
4 days ago · News release. Ultragenyx Pharmaceutical Inc. December 19, 2024. Accessed January 7, ... (CSF) correlates with improved long-term cognitive function in patients with mucopolysaccharidosis type IIIA (MPS IIIA) following treatment with UX111 gene therapy. Presented at: WorldSymposium; February 4-9; San Diego, California.
Global Mucopolysaccharidosis Treatment Market to Witness …
NEWARK, Del, Jan. 02, 2025 (GLOBE NEWSWIRE) -- The global mucopolysaccharidosis treatment market, valued at USD 2,669.1 million in 2024, is estimated to grow at a compound annual growth rate...
Mucopolysaccharidoses (MPS) - Johns Hopkins Medicine
Mucopolysaccharidoses is a group of genetic disorders — Hurler; Hunter; Sanfilippo A, B, C and D; Morquio A, B and C; and Maroteaux–Lamy — characterized by the excretion of mucopolysaccharide (sugar) in the urine.
Current and new therapies for mucopolysaccharidoses
Feb 1, 2023 · In this review, we discuss the basic principle underlying MPS treatment (enzymatic “cross correction”), and we review the three general modalities currently available: hematopoietic stem cell transplantation, enzymatic replacement, and gene therapy.
Hematopoietic stem cell transplantation in children with ...
Oct 14, 2024 · Mucopolysaccharidosis IVA (MPS IVA; Morquio syndrome) is a lysosomal storage disorder and features systemic skeletal dysplasia that is caused by defective Nacetylgalactosamine-6-sulfate sulfatase...
Mucopolysaccharidosis Type I: Current Treatments, Limitations, …
Mucopolysaccharidosis type I (MPS I) is a lysosomal disease, caused by a deficiency of the enzyme alpha-L-iduronidase (IDUA). Currently approved treatments consist of enzyme replacement therapy (ERT) and/or hematopoietic stem cell transplantation (HSCT).
Mucopolysaccharidoses - Symptoms, Causes, Treatment - NORD
Jun 23, 2017 · In individuals with MPS disorders, deficiency or malfunction of specific lysosomal enzymes leads to an abnormal accumulation of certain complex carbohydrates (mucopolysaccharides or glycosaminoglycans) in the arteries, skeleton, eyes, …