26 studies found for:    "ankyloblepharon-ectodermal defects-cleft lip palate syndrome" OR "Ectodermal Dysplasias"
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Rank Status Study
21 Completed Study of TD101, a Small Interfering RNA (siRNA) Designed for Treatment of Pachyonychia Congenita
Condition: Pachyonychia Congenita
Interventions: Drug: TD101;   Drug: Normal saline (placebo)
22 Recruiting Topical Sirolimus for Plantar Keratoderma in Adults With Pachyonychia Congenita (PC)
Conditions: Pachyonychia Congenita;   Plantar Keratoderma
Intervention: Drug: Topical Sirolimus
23 Completed Natural History of Asphyxiating Thoracic Dystrophy (DTJ)
Condition: Asphyxiating Thoracic Dystrophy
Intervention: Genetic: IFT80 screening
24 Recruiting Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)
Condition: Pachyonychia Congenita
Interventions: Drug: 1% sirolimus cream (TD201 1%);   Drug: placebo cream (vehicle control)
25 Recruiting Study of Selected X-linked Disorders: Goltz Syndrome
Conditions: Focal Dermal Hypoplasia (FDH);   Goltz Syndrome;   X Linked Disorders
Intervention:
26 Unknown  Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita
Condition: Epidermolysis Bullosa Simplex
Interventions: Drug: Dysport® (Botulinumtoxin A (Btx A));   Drug: Placebo

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Indicates status has not been verified in more than two years