137 studies found for:    "PPM-X 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 Recruiting Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome
Conditions: Fragile X Syndrome;   Genetic Diseases
Interventions: Drug: Lovastatin;   Drug: Placebo
3 Not yet recruiting Triac Trial II in MCT8 Patients
Condition: Allan-Herndon-Dudley Syndrome
Intervention: Drug: Triac
4 Active, not recruiting Triac Trial in MCT8 Patients
Condition: Allan-Herndon-Dudley Syndrome
Intervention: Drug: Triac
5 Completed Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW
Conditions: Rett Syndrome;   Prader-Willi Syndrome;   Angelman Syndrome;   Sleep Problems
Intervention:
6 Not yet recruiting Study of Idursulfase-beta (GC1111) in Hunter Syndrome
Condition: Mucopolysaccharidosis II
Interventions: Biological: idursulfase beta;   Biological: idursulfase
7 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
8 Not yet recruiting Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:
Condition: Pyruvate Dehydrogenase Complex Deficiency
Interventions: Drug: Dichloroacetate (DCA);   Other: Placebo;   Genetic: Genotype
9 Not yet recruiting A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy
Condition: X-linked Adrenoleukodystrophy
Intervention: Dietary Supplement: vitamin D3
10 Recruiting Pharmacological Treatment of Rett Syndrome With Statins
Condition: Rett Syndrome
Intervention: Drug: Lovastatin
11 Not yet recruiting An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome
Condition: Rett Syndrome
Intervention: Procedure: Intravenous infusion
12 Recruiting Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy
Conditions: Metachromatic Leukodystrophy;   Adrenoleukodystrophy
Intervention: Genetic: transduced CD34+ hematopoietic stem cell
13 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
14 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
15 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
16 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
17 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
18 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
19 Withdrawn MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)
Condition: Cerebral Adrenoleukodystrophy
Intervention: Biological: Mesenchymal Stem Cells
20 Withdrawn Analysis of the Glutathione Cycle in Children With Rett Syndrome
Condition: Rett Syndrome
Intervention:

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Indicates status has not been verified in more than two years