FRESH-PEF74146-draft-en
RaDiCo-MPS - Mucopolysaccharidosis patients in France in the era of specific therapeutics
RaDiCo-MPS
| Name | Country code |
|---|---|
| France | fr |
['Clinical data','Biological data','Participant-reported health data']
Individus
| Topic | Vocabulary |
|---|---|
| Mucopolysaccharidosis | cim-11 |
{
"level_sex_clusion_I": [
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D005260"
},
"value": "Female"
},
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D008297"
},
"value": "Male"
}
],
"level_age_clusion_I": [
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D007231"
},
"value": "Infant, Newborn (birth to 28 days)"
},
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D007223"
},
"value": "Infant (28 days to 2 years)"
},
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D002675"
},
"value": "Child, Preschool (2 to 5 years)"
},
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D002648"
},
"value": "Child (6 to 12 years)"
},
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D055815"
},
"value": "Young Adult (19 to 24 years)"
},
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D000328"
},
"value": "Adult (25 to 44 years)"
},
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D008875"
},
"value": "Middle Aged (45 to 64 years)"
}
],
"level_type_clusion_I": "Patients population",
"level_type_clusion_other": "",
"clusion_I": "The RaDiCo-MPS Cohort inclusion criteria are the following: • Confirmed diagnosis of MPS based on clinically relevant enzyme deficiency, with abnormally elevated GAG urinary excretion and\/or identification of pathogenic mutations. • Signed informed consent or parents\/guardian non-opposition for deceased patients (minor or protected major)",
"clusion_E": "There are no non-inclusion criteria."
}
| Name |
|---|
| Thierry;BILLETTE DE VILLEMEUR |
| Name | Role |
|---|---|
| INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) | sponsor |
| Name |
|---|
| ; |
| Agency name |
|---|
| CPP |
| CESREES |
['Through organizations (health services or institutions, schools, businesses, etc.)']
['{"concept":{"vocab":"CESSDA","vocabURI":"Other"},"value":"Other"}']
Access on specific project only
Observational Study
| Start | End |
|---|---|
| 2017-01-01 | 3000-01-01 |
| Standard |
|---|
| ['Drug dictionary (DCIs)'] |
['Data Management Plan and Data Validation Plan. Continuous data management (automatic control rules and query system)']
Requests for access to RaDiCo-MPS data (aggregated or individual) will be considered by the Scientific Committee following the submission of a summary of a specific research project, as defined in the Charter of access to resources.
{"extLink":{"title":"COAR","uri":"http://purl.org/coar/access_right/c_16ec"},"value":"Restricted access"}
FRESH-PEF74146-draft-en
| Name | Affiliation |
|---|---|
| Elodie Mercier | INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) |