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155 studies found for:    "PPM-X syndrome" OR "Mental Retardation, X-Linked"
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Rank Status Study
21 Recruiting Combined Treatment of Minocycline and Lovastatin to Treat Individuals With Fragile X Syndrome
Condition: Fragile X Syndrome
Interventions: Drug: Minocycline, then Minocycline/Lovastatin;   Drug: Lovastatin, then Minocycline/Lovastatin
22 Completed Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW
Conditions: Rett Syndrome;   Prader-Willi Syndrome;   Angelman Syndrome;   Sleep Problems
23 Not yet recruiting Study of Idursulfase-beta (GC1111) in Hunter Syndrome
Condition: Mucopolysaccharidosis II
Interventions: Biological: idursulfase beta;   Biological: idursulfase
24 Recruiting Effects of Social Gaze Training on Brain and Behavior in Fragile X Syndrome
Conditions: Fragile X Syndrome;   Intellectual Disability
Intervention: Behavioral: Social gaze training
25 Not yet recruiting Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:
Condition: Pyruvate Dehydrogenase Complex Deficiency
Interventions: Drug: Dichloroacetate (DCA);   Other: Placebo;   Genetic: Genotype
26 Recruiting A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy
Condition: X-linked Adrenoleukodystrophy
Intervention: Dietary Supplement: vitamin D3
27 Completed Pharmacological Treatment of Rett Syndrome With Statins
Condition: Rett Syndrome
Intervention: Drug: Lovastatin
28 Active, not recruiting An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome
Condition: Rett Syndrome
Intervention: Procedure: Intravenous infusion
29 Recruiting Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy
Conditions: Metachromatic Leukodystrophy;   Adrenoleukodystrophy
Intervention: Genetic: transduced CD34+ hematopoietic stem cell
30 Recruiting Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases
Conditions: Spinal Muscular Atrophy;   Charcot-Marie-Tooth Disease;   Muscular Dystrophy;   Spinal Muscular Atrophy With Respiratory Distress 1;   Amyotrophic Lateral Sclerosis;   Motor Neuron Disease;   Neuromuscular Disease;   Peroneal Muscular Atrophy;   Fragile X Syndrome
Intervention: Other: sample collection
31 Recruiting Decisional Capacity and Informed Consent in Fragile X Syndrome
Condition: Fragile X Syndrome (FXS)
Interventions: Other: Paper-Based Informed Consent;   Device: Tablet-Based Consent
32 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
33 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
34 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
35 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
36 Withdrawn MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)
Condition: Cerebral Adrenoleukodystrophy
Intervention: Biological: Mesenchymal Stem Cells
37 Withdrawn Analysis of the Glutathione Cycle in Children With Rett Syndrome
Condition: Rett Syndrome
38 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
39 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
40 Unknown  Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy
Condition: X-linked Adrenoleukodystrophy
Intervention: Drug: Lorenzo's Oil

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Indicates status has not been verified in more than two years