Medical Trials Mps

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Clinical Trials - MPS Society

https://mpssociety.org/learn/treatments/clinical-trials/

Clinical trials are research studies that determine whether treatments or devices are safe for humans. These studies also look for effective medical approaches for MPS and ML and help provide reliable data for patients, …

Mucopolysaccharidoses | National Institute of …

https://www.ninds.nih.gov/health-information/disorders/mucopolysaccharidoses

MPS IIID is caused by the missing or deficient enzyme N-acetylglucosamine-6-sulfate; MPS IV (also known as Morquio syndrome) has two subtypes that result from …

Mucopolysaccharidoses (MPS) | ASGCT - American Society of …

https://patienteducation.asgct.org/disease-treatments/mps

Mucopolysaccharidoses (MPS) Home > Disease Treatments > Mucopolysaccharidoses (MPS) Mucopolysaccharidoses (MPS) are a group of genetic disorders that affect our body’s ability to break down and …

Open-label Study of Anakinra in MPS III - Full Text View ...

https://clinicaltrials.gov/ct2/show/NCT04018755

Sanfilippo syndrome, or mucopolysaccharidosis type III (MPS III), is a disorder of metabolism, associated with insufficient production of a lysosomal enzyme needed for …

Mucopolysaccharidosis I (MPS I) Registry - Full Text …

https://clinicaltrials.gov/ct2/show/NCT00144794

The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MPS …

Clinical Trials | University of Miami Health System

https://umiamihealth.org/clinical-trials

Clinical trials are research studies that involve people. They are the final step in a long process that begins with research that is done in a lab. Clinical trials are key to …

Mucopolysaccharidosis (MPS VI) Clinical Trials - BioMarin

https://www.biomarin.com/clinical-trials/mps-vi/

MPS VI (mucopolysaccharidosis VI), also known as Maroteaux-Lamy Syndrome, is an inherited lysosomal storage disorder caused by the deficiency of N-acetylgalactosamine 4-sulfatase (arylsulfatase B), an …

Unfavourable results from medical trials are being …

https://www.theguardian.com/science/2013/sep/17/medical-trial-results-withheld-mps-report

Drug companies and medical researchers are putting patients' lives in danger by failing to publish unfavourable results from clinical trials, MPs have warned. The lack of transparency means...

Mucopolysaccharidosis (MPS IVA) Clinical Trials - BioMarin

https://www.biomarin.com/clinical-trials/mps-iva/

mps iva Morquio A is a rare inherited disease that affects major organ systems in the body. The disease is a form of mucopolysaccharidosis, which is a type of lysosomal storage disorder.

MPS VII - MPS Society

https://mpssociety.org/learn/diseases/mps-vii/

MPS VII is a mucopolysaccharide disease also known as Sly syndrome. It takes its name from Dr. William Sly who originally described the condition in 1972. What causes this disease? Mucopolysaccharides are chains of …

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