128 studies found for:    "Renpenning syndrome" OR "Mental Retardation, X-Linked"
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Rank Status Study
1 Enrolling by invitation Genetic Disease Gene Identification
Conditions: Congenital Vertical Talus;   Familial Encephalopathy With Neuroserpin Inclusion Bodies;   Idiopathic Generalised Epilepsy;   Familial Dementia;   X-Linked Mental Retardation
Intervention:
2 Not yet recruiting Triac Trial II in MCT8 Patients
Condition: Allan-Herndon-Dudley Syndrome
Intervention: Drug: Triac
3 Recruiting Triac Trial in MCT8 Patients
Condition: Allan-Herndon-Dudley Syndrome
Intervention: Drug: Triac
4 Not yet recruiting Fragile X Syndrome Decision Aid
Condition: Fragile X Syndrome (FXS)
Interventions: Other: Paper-Based Informed Consent;   Device: Tablet-Based Consent
5 Not yet recruiting Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age
Condition: Hunter Syndrome
Intervention: Drug: Elaprase for intravenous (IV) infusion
6 Recruiting Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI
Conditions: Mucopolysaccharidosis Type I;   Mucopolysaccharidosis Type II;   Mucopolysaccharidosis Type VI
Interventions: Drug: Adalimumab;   Other: Placebo
7 Not yet recruiting Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions
Conditions: Osteopenia;   Spinal Muscular Atrophy;   Cerebral Palsy;   Muscular Dystrophy;   Spina Bifida;   Rett Syndrome
Intervention: Other: Assisted Standing Treatment Program
8 Enrolling by invitation Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Patients With Hunter Syndrome That Have Completed Study HGT-HIT-094
Condition: Hunter Syndrome
Intervention: Drug: Idursulfase-IT
9 Not yet recruiting MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)
Condition: Cerebral Adrenoleukodystrophy
Intervention: Biological: Mesenchymal Stem Cells
10 Not yet recruiting Analysis of the Glutathione Cycle in Children With Rett Syndrome
Condition: Rett Syndrome
Intervention:
11 Recruiting Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome
Condition: Mucopolysaccharidosis II
Intervention: Biological: AGT-182
12 Recruiting UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Conditions: Adrenoleukodystrophy;   Batten Disease;   Mucopolysaccharidosis II;   Leukodystrophy, Globoid Cell;   Leukodystrophy, Metachromatic;   Neimann Pick Disease;   Pelizaeus-Merzbacher Disease;   Sandhoff Disease;   Tay-Sachs Disease;   Brain Diseases, Metabolic, Inborn
Intervention: Biological: DUOC-01
13 Available Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy
Condition: X-linked Adrenoleukodystrophy
Intervention: Drug: Lorenzo's Oil
14 Recruiting Observational Safety and Efficacy Study of Allo-HSCT to Treat Childhood Cerebral Adrenoleukodystrophy
Condition: X-linked Adrenoleukodystrophy Cerebral Childhood
Intervention: Genetic: Allo-HSCT
15 Not yet recruiting Citocoline for Treatment of FXTAS
Condition: Balance and Cognitive Deficits in Fragile X-associated Tremor Ataxia Syndrome
Intervention: Drug: citocoline
16 Recruiting MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Conditions: Mucopolysaccharidosis I;   Mucopolysaccharidosis II;   Mucopolysaccharidosis VI;   Mucopolysaccharidosis VII;   Hurler Syndrome;   Hunter Syndrome;   Maroteaux Lamy Syndrome;   Sly Syndrome;   Glycoprotein Metabolic Disorders;   Alpha Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Adrenoleukodystrophy;   Peroxisomal Disorders;   Osteopetrosis;   Sphingolipidosis;   Gangliosidosis;   Globoid Cell Leukodystrophy;   Metachromatic Leukodystrophy;   Niemann Pick B;   Niemann Pick C Subtype 2;   I-cell Disease
Interventions: Procedure: blood stem cell transplant;   Drug: Rabbit Anti-Thymocyte Globulin (ATG);   Drug: Fludarabine;   Drug: Busulfan;   Drug: Cyclophosphamide;   Drug: Cyclosporine A (CSA);   Drug: Methylprednisolone;   Drug: Mycophenolate Mofetil (MMF);   Drug: Granulocyte-Colony Stimulating Factor (G-CSF);   Drug: Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF);   Drug: N-acetylcysteine;   Drug: Celecoxib;   Drug: Vitamin E;   Drug: Alpha Lipoic Acid
17 Active, not recruiting Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)
Condition: Rett Syndrome
Intervention: Drug: Glatiramer Acetate
18 Active, not recruiting A 6-week, Study of MG01CI Low Dose and High Dose Compared With Placebo in Adults and Adolescents With Fragile X Syndrome
Condition: Fragile X Syndrome
Intervention: Drug: MG01CI extended-release tablet
19 Recruiting Osteoporosis in RETT Syndrome
Condition: RETT Syndrome With Proven MECP2 Mutation
Intervention: Other: biological markers and evaluation of the mineral density at the lumber spine using DEXA
20 Terminated Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)
Conditions: Mucopolysaccharidosis;   Mucopolysaccharidosis I;   Mucopolysaccharidosis II;   Mucopolysaccharidosis VI
Intervention:

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Indicates status has not been verified in more than two years