Gene Therapy in Patients With Mucopolysaccharidosis Disease
Mucopolysaccharidosis Type VIThis study investigated the safety and efficacy of gene therapy approaches for Mucopolysaccharidosis type VI disease caused by the deficiency of arylsulfatase B (ARSB) enzyme. The aim of the study is to evaluate the safety and efficacy of the treatment.
A Treatment Extension Study of Mucopolysaccharidosis Type IIIB
Mucopolysaccharidosis Type IIIBMPS III BThe primary objectives of this study are to evaluate the long-term safety and tolerability of AX 250 administered to subjects with MPS IIIB by an implanted ICV reservoir and catheter and to evaluate the impact of long-term AX 250 treatment on cognitive function in patients with MPS IIIB as assessed by developmental quotient (DQ).
An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Mucopolysaccharidosis IIMulticenter, open-label, single-group, designed to evaluate the long term efficacy and safety of study drug for the treatment of the MPS II.
Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis...
Mucopolysaccharidosis Type IIIAPatients with MPS IIIA have a clinical disorder marked by severe and progressive brain disease and neurological symptoms due to the accumulation of undigested glycosaminoglycans in all cells of the body. This study will be the first in human clinical trial to explore the safety, tolerability and clinical efficacy of ex vivo gene therapy (autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene) in MPSIIIA patients. Following treatment with the gene therapy patients will be followed up for a minimum of 3 years.
Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and...
Mucopolysaccharidosis IV AMucopolysaccharidosis VI7 moreMucopolysaccharidoses (MPS) are multisystemic diseases with significant clinical overlap between their types, with cardiac problems being among the most commonly observed manifestations and are also among the main causes of mortality in these patients. For some of the cardiovascular manifestations, such as aortic root dilation and valve diseases, there is no effective treatment currently available. Losartan, on the other hand, has been shown to be an effective drug for dilation of the aortic root, at least in animal models. This study aims to evaluate the safety and efficacy of losartan in patients with MPS VI and other mucopolysaccharidoses.
An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
Mucopolysaccharidosis IIA Phase II open-label, parallel group, 2 sites (Brazil), designed to evaluate the long term safety and efficacy of study drug for the treatment of the MPS II.
Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With...
Mucopolysaccharidosis IHThis is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human α-L-iduronidase gene for the treatment of patients affected by Mucopolysaccharidosis Type I, Hurler variant
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
MPS IIIASanfilippo Syndrome2 moreThe main objective of this study is to evaluate the efficacy and safety of ABO-102 for the treatment of MPS IIIA.
A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102
Mucopolysaccharidosis III-AThe main objective of this study is to evaluate the long-term safety and tolerability of ABO-102 in participants with MPS IIIA.
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Mucopolysaccharidosis IMucopolysaccharidosis II6 moreThis is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with LSDs.