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A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa
This is a 78-week (approximately a year and a half) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 6 months and older, who have been diagnosed with Dystrophic Epidermolysis Bullosa (DEB). Participants will be dosed weekly with the topical B-VEC therapy. The primary endpoint will be to assess long term safety and tolerability of the topical gene therapy. The study is for those who participated in Phase III study, as well as, new participants who were unable to participate in the Phase III study, who meet all enrollment criteria.
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A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis Bullosa
To determine if Serlopitant (when taken by mouth) is safe and works on itch in patients aged 13 and above with EB.
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An Open-Label and Long-Term Extension Study to Evaluate the Efficacy and Safety of Ustekinumab in the Treatment of Patients With Ichthyoses
The ichthyoses are a group of lifelong genetic disorders that share characteristics of generalized skin thickening, scaling and underlying cutaneous inflammation. The vast majority are orphan disorders and are associated with extremely poor quality of life related to social ostracism from altered appearance, associated itchiness and discomfort, and functional limitations from the skin disease. Among the more common "orphan" forms of ichthyosis are autosomal recessive congenital ichthyosis (ARCI; includes lamellar ichthyosis/LI and congenital ichthyosiform erythroderma/CIE), Netherton syndrome (NS) and epidermolytic ichthyosis (EI)....
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An Open-label, Single-dose Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Brodalumab in Pediatric Subjects
An Open-label, Single-dose Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Brodalumab in Pediatric Subjects
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A Pilot Study of Hemoporfin PDT in Children(2-7 Years Old) With Port-wine Stain
This pilot study aims to evaluate the safety and efficacy of hemoporfin photodynamic therapy (PDT) with different light doses for port-wine stain (PWS）in 2-7 years old children. The pharmacokinetic behavior and pharmacokinetic parameters of hemoporfin in children will be investigated as well.
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A Pilot Study to Explore the Role of Gut Flora in Acne
This study seeks to correlate microbiome sequencing data with information provided by patients and their medical records.
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A Pilot Study to Explore the Role of Gut Flora in Epidermolysis Bullosa
This study seeks to correlate microbiome sequencing data with information provided by patients and their medical records.
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A Pilot Study to Explore the Role of Gut Flora in Lupus
This study seeks to correlate microbiome sequencing data with information provided by patients and their medical records.
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Application of MRI for Musculoskeletal Involvement in SLE
Articular involvement can reach up to 95% within the chronic multisystemic manifestations of SLE (1). Originally, a non-erosive pattern of articular inflammation was described, but the emergence of more sensitive imaging techniques, such as MRI (2, 3), show synovitis, erosions (hand: 47-48%, carpus 82-84% in SLE; and hand: 18%, carpus 97% in healthy individuals), bone oedema (hand: 4-5%, carpus 13-16% in SLE; and 0% in healthy individuals) and tenosynovitis (hand 47%, carpus 79%; not evaluated in healthy individuals) in patients with SLE (4, 5). Nowadays, a specific validated pattern of articular involvement associated with this disease ...
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Apremilast Pregnancy Exposure Registry
The purpose of the Apremilast Pregnancy Exposure Registry is to monitor planned and unplanned pregnancies exposed to apremilast and to evaluate the safety of this medication relative to specified pregnancy outcomes, and to evaluate potential effects of prenatal apremilast exposure on infant health status through one year of age.
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