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Found 23 clinical trials
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Ophthalmic Manifestations of Patients With Mucopolysaccharidosis

Mucopolysaccharidosis (MPSs) are a group of disorders caused by inherited defects in lysosomal enzymes resulting in widespread intra- and extra-cellular accumulation of glycosaminoglycan(1,2

  • 0 views
  • 06 Mar, 2021
  • 1 location
None
Respiratory Cathepsins Proteases Inhibitors and Glycosaminoglycans (GAG) in Mucopolysaccharidosis

Mucopolysaccharidosis (MPS) are a group of inherited, metabolic diseases caused by a deficiency of lysosomal enzymes that degrade glycosaminoglycans (GAGs). Loss of their activity results in

  • 0 views
  • 09 May, 2021
  • 7 locations
None
The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients

The objective of this study is to evaluate the long term safety and efficacy of once weekly dosing of idurasulfase-beta 0.5mg/kg administered in Hunter Syndrome(Mucopolysaccharidosis II

mucopolysaccharidosis
idursulfase
  • 108 views
  • 07 Nov, 2020
None
A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101

This is a multicenter, non-interventional, long-term follow-up (LTFU) study in participants who have been treated with ABO-101 in a prior trial. Eligible participants will undergo clinical evaluations at prespecified intervals for 3 years from the last visit in the prior clinical trial (up to 5 years post-treatment).

  • 0 views
  • 06 Mar, 2021
  • 3 locations
None
Mucopolysaccharidosis VII Disease Monitoring Program

The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.

  • 0 views
  • 08 Dec, 2021
  • 12 locations
None
Mucopolysaccharidosis I (MPS I) Registry

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 I Registry will provide

  • 0 views
  • 05 Nov, 2021
  • 170 locations
None
Mucopolysaccharidosis I (MPS I) Registry

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 I Registry will provide

  • 398 views
  • 04 Nov, 2021
  • 211 locations
None
A Multicenter Multinational Observational Morquio A Registry Study (MARS)

The objectives of this program are: to characterize and describe the Mucopolysaccharidosis IV type A (MPS IVA) population as a whole, including the heterogeneity, progression, and natural

  • 140 views
  • 07 Aug, 2021
  • 94 locations
None
A Study of Potential Treatment-Responsive Biomarkers in Hunter Syndrome

This is an observational, Pre-phase 1 study of biomarkers in patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.

ids gene
mucopolysaccharidosis
anesthesia
general anesthesia
  • 202 views
  • 11 Nov, 2021
  • 8 locations
None
Longitudinal Study of Neurodegenerative Disorders

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.

cell transplantation
palliative care
stem cell transplantation
genetic disorders
neurodegenerative disorders
  • 359 views
  • 13 Nov, 2021
  • 1 location