xml: <clinical_study>
    <!-- This xml conforms to an XML Schema at:
      https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
    <required_header>
      <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
      <link_text>Link to the current ClinicalTrials.gov record.</link_text>
      <url>https://clinicaltrials.gov/ct2/show/NCT06036693</url>
    </required_header>
    <id_info>
      <org_study_id>C16-53</org_study_id>
      <nct_id>NCT06036693</nct_id>
    </id_info>
    <brief_title>MPS (RaDiCo Cohort) (RaDiCo-MPS)</brief_title>
    <official_title>Mucopolysaccharidosis Patients in France in the Era of Specific Therapeutics</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Institut National de la Santé Et de la Recherche Médicale, France</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Institut National de la Santé Et de la Recherche Médicale, France</source>
    <oversight_info>
      <has_dmc>No</has_dmc>
      <is_fda_regulated_drug>No</is_fda_regulated_drug>
      <is_fda_regulated_device>No</is_fda_regulated_device>
    </oversight_info>
    <brief_summary>
      <textblock>
        The goal of this observational study is to characterize the epidemiology and natural history&#xD;
        of MPS diseases by building a retrospective and prospective collection of extensive&#xD;
        phenotypic data from French MPS patients.&#xD;
      </textblock>
    </brief_summary>
    <overall_status>Recruiting</overall_status>
    <start_date type="Actual">December 20, 2017</start_date>
    <completion_date type="Anticipated">December 20, 2024</completion_date>
    <primary_completion_date type="Anticipated">December 20, 2024</primary_completion_date>
    <study_type>Observational</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <observational_model>Cohort</observational_model>
      <time_perspective>Other</time_perspective>
    </study_design_info>
    <primary_outcome>
      <measure>Evaluation of the clinical data of MPS like growth for each system</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the clinical data of MPS like signs for each system</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the clinical data of MPS like symptoms for each system</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the clinical data of MPS like complications for each system</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the clinical data of MPS like psychomotor milestones</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the clinical data of MPS like cognitive evolution</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the clinical data of MPS like handicap using scales adapted to multivisceral disease for all types of MPS</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the clinical data of MPS like handicap using scales adapted to cognitive and neurologic disease for the types I, II, III VII</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the radiological data of MPS like standard bone radiographs</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the radiological data of MPS like abdominal echography</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the radiological data of MPS like echocardiography</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the radiological data of MPS like cerebral and medullar tomodensitometry</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the radiological data of MPS like magnetic resonance imaging</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the electrophysiological data of MPS like EMG</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the electrophysiological data of MPS like EEG</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the electrophysiological data of MPS like ERG</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the biochemical data of MPS like urinary GAG before specific treatment</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the biochemical data of MPS like urinary GAG during specific treatment</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the biochemical data of MPS like enzyme activities before specific treatment</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the biochemical data of MPS like enzyme activities during specific treatment</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the biochemical data of MPS like specific antibodies</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Evaluation of the molecular data of MPS</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </primary_outcome>
    <secondary_outcome>
      <measure>Description of the management of MPS diseases without specific treatment</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Description of the management of MPS diseases before specific treatment</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Description of the management of MPS diseases under specific treatment.</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Description of the outcome of MPS diseases without specific treatment</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Description of the outcome of MPS diseases before specific treatment</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Description of the outcome of MPS diseases under specific treatment.</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Identification of mutation(s) in each MPS type</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Establishment of genotype/phenotype relationships in each MPS type.</measure>
      <time_frame>Through study completion, an average of 5 years</time_frame>
    </secondary_outcome>
    <enrollment type="Anticipated">1000</enrollment>
    <condition>Mucopolysaccharidosis I</condition>
    <condition>Mucopolysaccharidosis II</condition>
    <condition>Mucopolysaccharidosis III</condition>
    <condition>Mucopolysaccharidosis IV</condition>
    <condition>Mucopolysaccharidosis VI</condition>
    <condition>Mucopolysaccharidosis VII</condition>
    <condition>Mucopolysaccharidosis IX</condition>
    <condition>Multiple Sulfatase Deficiency Disease</condition>
    <eligibility>
      <study_pop>
        <textblock>
          Prevalent and incident patients will be included in the cohort RaDiCo-MPS.&#xD;
        </textblock>
      </study_pop>
      <sampling_method>Non-Probability Sample</sampling_method>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  Confirmed diagnosis of MPS based on clinically relevant enzyme deficiency, with&#xD;
               abnormally elevated GAG urinary excretion and/or identification of pathogenic&#xD;
               mutations.&#xD;
  &#xD;
            -  Signed informed consent or parents/guardian non-opposition for deceased patients&#xD;
               (minor or protected major)&#xD;
  &#xD;
          There are no non-inclusion criteria.&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>N/A</minimum_age>
      <maximum_age>N/A</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_official>
      <last_name>Thierry BILLETTE DE VILLEMEUR</last_name>
      <role>Principal Investigator</role>
      <affiliation>INSERM UMR 1141</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Bénédicte HERON</last_name>
      <phone>01 44 73 65 75</phone>
      <email>benedicte.heron@aphp.fr</email>
    </overall_contact>
    <location>
      <facility>
        <name>Centre Hospitalier Universitaire d'Angers</name>
        <address>
          <city>Angers</city>
          <country>France</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Magalie BARTH</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital des Enfants - Groupe Hospitalier Pellegrin</name>
        <address>
          <city>Bordeaux</city>
          <country>France</country>
        </address>
      </facility>
      <status>Not yet recruiting</status>
      <contact>
        <last_name>Didier LACOMBE</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital Morvan</name>
        <address>
          <city>Brest</city>
          <country>France</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Elise SACAZE</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital d'Estaing</name>
        <address>
          <city>Clermont-Ferrand</city>
          <country>France</country>
        </address>
      </facility>
      <status>Not yet recruiting</status>
      <contact>
        <last_name>Marc BERGER</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital Beaujon</name>
        <address>
          <city>Clichy</city>
          <country>France</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Nadia BELMATOUG</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital Raymond-Poincaré</name>
        <address>
          <city>Garches</city>
          <country>France</country>
        </address>
      </facility>
      <status>Not yet recruiting</status>
      <contact>
        <last_name>Dominique-Paul GERMAIN</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital Jeanne de Flandre</name>
        <address>
          <city>Lille</city>
          <country>France</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Anne-Sophie GUEMANN</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital de la Timone</name>
        <address>
          <city>Marseille</city>
          <country>France</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Brigitte CHABROL</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital Gui de Chauliac</name>
        <address>
          <city>Montpellier</city>
          <country>France</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Agathe ROUBERTIE</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital Brabois</name>
        <address>
          <city>Nancy</city>
          <country>France</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>François FEILLET</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital Armand Trousseau</name>
        <address>
          <city>Paris</city>
          <country>France</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Bénédicte HERON</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital de la Croix Saint-Simon</name>
        <address>
          <city>Paris</city>
          <country>France</country>
        </address>
      </facility>
      <status>Not yet recruiting</status>
      <contact>
        <last_name>Wladimir MAUHIN</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital de la Pitié-Salpêtrière</name>
        <address>
          <city>Paris</city>
          <country>France</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Yann NADJAR</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital Necker-Enfants Malades</name>
        <address>
          <city>Paris</city>
          <country>France</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Anaïs BRASSIER</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital Robert Debré</name>
        <address>
          <city>Paris</city>
          <country>France</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Samia PICHARD</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Centre Hospitalier de Pau</name>
        <address>
          <city>Pau</city>
          <country>France</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Pierre BEZE-BEYRIE</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>American Memorial Hospital</name>
        <address>
          <city>Reims</city>
          <country>France</country>
        </address>
      </facility>
      <status>Not yet recruiting</status>
      <contact>
        <last_name>Nathalie BEDNAREK</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital Pontchaillou</name>
        <address>
          <city>Rennes</city>
          <country>France</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Bérengère CADOR</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital Charles Nicolle</name>
        <address>
          <city>Rouen</city>
          <country>France</country>
        </address>
      </facility>
      <status>Not yet recruiting</status>
      <contact>
        <last_name>Stéphanie TORRE</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital de Hautepierre</name>
        <address>
          <city>Strasbourg</city>
          <country>France</country>
        </address>
      </facility>
      <status>Not yet recruiting</status>
      <contact>
        <last_name>Marie-Thérèse ABI WARDE</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Clinique Monié</name>
        <address>
          <city>Toulouse</city>
          <country>France</country>
        </address>
      </facility>
      <status>Not yet recruiting</status>
      <contact>
        <last_name>Francis GACHES</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital des Enfants</name>
        <address>
          <city>Toulouse</city>
          <country>France</country>
        </address>
      </facility>
      <status>Not yet recruiting</status>
      <contact>
        <last_name>Guy TOUATI</last_name>
      </contact>
    </location>
    <location>
      <facility>
        <name>Hôpital Clocheville</name>
        <address>
          <city>Tours</city>
          <country>France</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Marine TARDIEU</last_name>
      </contact>
    </location>
    <location_countries>
      <country>France</country>
    </location_countries>
    <verification_date>July 2023</verification_date>
    <study_first_submitted>July 18, 2023</study_first_submitted>
    <study_first_submitted_qc>September 12, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">September 14, 2023</study_first_posted>
    <last_update_submitted>September 12, 2023</last_update_submitted>
    <last_update_submitted_qc>September 12, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">September 14, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Osteochondrodysplasias</mesh_term>
      <mesh_term>Mucopolysaccharidosis II</mesh_term>
      <mesh_term>Multiple Sulfatase Deficiency Disease</mesh_term>
      <mesh_term>Mucopolysaccharidoses</mesh_term>
      <mesh_term>Mucopolysaccharidosis I</mesh_term>
      <mesh_term>Mucopolysaccharidosis VI</mesh_term>
      <mesh_term>Mucopolysaccharidosis IV</mesh_term>
      <mesh_term>Mucopolysaccharidosis III</mesh_term>
      <mesh_term>Mucopolysaccharidosis VII</mesh_term>
      <mesh_term>Deficiency Diseases</mesh_term>
    </condition_browse>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: download date: ClinicalTrials.gov processed this data on September 20, 2023
  link text: Link to the current ClinicalTrials.gov record.
  url: https://clinicaltrials.gov/ct2/show/NCT06036693
  org study id: C16-53
  nct id: NCT06036693
  lead sponsor: 
  has dmc: No
  is fda regulated drug: No
  is fda regulated device: No
  The goal of this observational study is to characterize the epidemiology and natural history
        of MPS diseases by building a retrospective and prospective collection of extensive
        phenotypic data from French MPS patients.
  observational model: Cohort
  time perspective: Other
  measure: Evaluation of the clinical data of MPS like growth for each system
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the clinical data of MPS like signs for each system
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the clinical data of MPS like symptoms for each system
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the clinical data of MPS like complications for each system
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the clinical data of MPS like psychomotor milestones
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the clinical data of MPS like cognitive evolution
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the clinical data of MPS like handicap using scales adapted to multivisceral disease for all types of MPS
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the clinical data of MPS like handicap using scales adapted to cognitive and neurologic disease for the types I, II, III VII
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the radiological data of MPS like standard bone radiographs
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the radiological data of MPS like abdominal echography
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the radiological data of MPS like echocardiography
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the radiological data of MPS like cerebral and medullar tomodensitometry
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the radiological data of MPS like magnetic resonance imaging
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the electrophysiological data of MPS like EMG
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the electrophysiological data of MPS like EEG
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the electrophysiological data of MPS like ERG
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the biochemical data of MPS like urinary GAG before specific treatment
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the biochemical data of MPS like urinary GAG during specific treatment
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the biochemical data of MPS like enzyme activities before specific treatment
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the biochemical data of MPS like enzyme activities during specific treatment
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the biochemical data of MPS like specific antibodies
  time frame: Through study completion, an average of 5 years
  measure: Evaluation of the molecular data of MPS
  time frame: Through study completion, an average of 5 years
  measure: Description of the management of MPS diseases without specific treatment
  time frame: Through study completion, an average of 5 years
  measure: Description of the management of MPS diseases before specific treatment
  time frame: Through study completion, an average of 5 years
  measure: Description of the management of MPS diseases under specific treatment.
  time frame: Through study completion, an average of 5 years
  measure: Description of the outcome of MPS diseases without specific treatment
  time frame: Through study completion, an average of 5 years
  measure: Description of the outcome of MPS diseases before specific treatment
  time frame: Through study completion, an average of 5 years
  measure: Description of the outcome of MPS diseases under specific treatment.
  time frame: Through study completion, an average of 5 years
  measure: Identification of mutation(s) in each MPS type
  time frame: Through study completion, an average of 5 years
  measure: Establishment of genotype/phenotype relationships in each MPS type.
  time frame: Through study completion, an average of 5 years
  study pop: 
  sampling method: Non-Probability Sample
  criteria: 
  gender: All
  minimum age: N/A
  maximum age: N/A
  healthy volunteers: No
  last name: Thierry BILLETTE DE VILLEMEUR
  role: Principal Investigator
  affiliation: INSERM UMR 1141
  last name: Bénédicte HERON
  phone: 01 44 73 65 75
  email: benedicte.heron@aphp.fr
  facility: 
  status: Recruiting
  contact: 
  facility: 
  status: Not yet recruiting
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  status: Recruiting
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  status: Not yet recruiting
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  status: Recruiting
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  status: Recruiting
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  status: Recruiting
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  status: Recruiting
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  status: Recruiting
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  status: Not yet recruiting
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  status: Recruiting
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  status: Not yet recruiting
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  status: Not yet recruiting
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  status: Not yet recruiting
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  status: Not yet recruiting
  contact: 
  facility: 
  status: Recruiting
  contact: 
  country: France
  responsible party type: Sponsor
  mesh term: Osteochondrodysplasias
  mesh term: Mucopolysaccharidosis II
  mesh term: Multiple Sulfatase Deficiency Disease
  mesh term: Mucopolysaccharidoses
  mesh term: Mucopolysaccharidosis I
  mesh term: Mucopolysaccharidosis VI
  mesh term: Mucopolysaccharidosis IV
  mesh term: Mucopolysaccharidosis III
  mesh term: Mucopolysaccharidosis VII
  mesh term: Deficiency Diseases