Abnormalities, Multiple - 435 Studies Found

Recruiting : Oxygen Versus Medical Air for Treatment of CSA in Prader Will Syndrome
:
  • Sleep Apnea, Central
  • Prader-Willi Syndrome

: 2017-01-20
: Biological: Medical Air vs Oxygen Medical Air/Oxygen will be given
Recruiting : Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study
:
  • Epidermolysis Bullosa Simplex
  • Epidermolysis Bullosa Simplex Kobner
  • Drug: Sirolimus, 2%
  • Drug: Vehicle

Recruiting : Setmelanotide Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity
:
  • Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic)
  • Leptin Receptor
    : 2017-01-03
    : Drug: Setmelanotide RM-493 once daily subcutaneous injection
Recruiting : Treatment of Sleep Disturbances in School-age Children With Down Syndrome
: Down Syndrome
: 2015-11-30
:
  • Behavioral: Behavioral Sleep Treatment

Recruiting : The Effects of Inhalational Anaesthetics in Cognitive Functions in Down Syndrome Patients
: Down Syndrome
: 2016-11-11
:
  • Drug: Sevoflurane
  • Drug: Desflurane
Completed : A Safety and Pharmakokinetic Study of A4250 Alone or in Combination With A3384
:
  • Orphan Cholestatic Liver Diseases
  • Primary Biliary Cirrhosis
  • Drug: A4250
  • Drug: CRC (A3384)
Enrolling by invitation : FibroScan™ in Pediatric Cholestatic Liver Disease Study Protocol
:
  • Biliary Atresia
  • Alagille Syndrome
  • Alpha1 Anti-Trypsin Defi
    : 2016-09-20
    : Other: Liver Stiffness Measurement (LSM) LSM will be measured via transient elastography utilizing the n
Recruiting : Ixazomib Citrate, Lenalidomide, and Dexamethasone in Treating Patients With POEMS Syndrome
:
  • Plasmacytoma
  • POEMS Syndrome

: 2016-09-26
:
  • Drug: Dexamethasone Given PO
Completed : Safety and Efficacy Study of NFC-1 in Subjects Aged 12-17 Years With 22q11.2DS & Associated Neuropsychiatric Conditions
: 22q11.2 Deletion Syndrome
: 2016-08-29
: Drug: NFC-1 Doses of NFC-1 will be administered as 50, 100, 200, or 400 mg twice daily as size 2, hard g
Active, not recruiting : Assessing the Feasibility of the Use of Visual Aids in Patient Education in Adults With Down Syndrome (DS)
: Down Syndrome
: 2016-08-27
:
  • Behavioral: Verbal instructions with picture diagram

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