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非在研适应症- |
最高研发阶段临床2期 |
首次获批国家/地区- |
首次获批日期- |
Natural History and Outcomes in X-Linked Hypohidrotic Ectodermal Dysplasia
The proposed natural history study will enroll male and female patients, ages 36 months and younger, who have a diagnosis of XLHED based on genetic testing and who have not received an investigational study drug. The study protocol will include collection of all relevant medical history and documentation of clinical outcomes using age-appropriate, minimally invasive technologies. Data will be collected both retrospectively, back to pregnancy assessments that may be available, and prospectively through age 5 yrs.
Extension Study of XLHED-Affected Male Subjects Treated With EDI200 in Protocol ECP-002
The goal of the ECP-002e extension study is to continue the evaluation of all EDI200-treated ECP-002 subjects up to age 10 yrs. No additional study drug administration is planned. The efficacy evaluations will incorporate growth and development parameters, frequency of infections and hospitalizations, and age-appropriate assessments of ectoderm-derived organ function. The safety evaluations will include physical examinations, adverse events and concomitant medication documentation, and laboratory testing. Funding Source - FDA OOPD
Phenotypic Properties in Individuals Affected With X-linked Hypohidrotic Ectodermal Dysplasia: Symptoms and Facial Recognition
The current study design incorporates two previously developed, non-invasive approaches to characterize the phenotype of individuals affected with XLHED.
100 项与 Edimer Pharmaceuticals, Inc. 相关的临床结果
0 项与 Edimer Pharmaceuticals, Inc. 相关的专利(医药)
2014-10-01·American Journal of Medical Genetics, Part A3区 · 生物学
Future developments in XLHED treatment approaches
3区 · 生物学
Review
作者: Huttner, Kenneth
100 项与 Edimer Pharmaceuticals, Inc. 相关的药物交易
100 项与 Edimer Pharmaceuticals, Inc. 相关的转化医学