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Rubin is a speaker for AbbVie, Amgen, Bayer, BMS, Castle Creek Biosciences, Celgene, Cutanea, Dermavant, DermTech, Galderma, Genentech, Helsinn, Incyte, LEO Pharma, Novartis, Ortho Dermatologics, Pfizer, Promius Pharma, Regeneron Pharmaceuticals Inc., Sanofi, Sun Pharma, and UCB; a consultant for AbbVie, Allergan, Amgen, Biofrontera, Castle Creek Biosciences, Celgene, Dermavant, DermTech, Incyte, LEO Pharma, Novartis, Ortho Dermatologics, Pfizer, Promius, Regeneron Pharmaceuticals Inc., Sanofi, and UCB; and an investigator for BMS, Cara Therapeutics, Castle Creek Biosciences, DermTech, Janssen, LEO Pharma, and Sanofi. Lara W. Lee is an advisory board member of Castle Creek Biosciences, Chiesi, Eli Lilly, Pfizer, and Regeneron Pharmaceuticals Inc.; a consultant for AbbVie, Amryt Pharma, Kimberly-Clark, Krystal Biotech, Novartis, and Pyramid Biosciences; an investigator for AbbVie, Amgen, Amryt Pharma, Arcutis Biotherapeutics, Castle Creek Biosciences, Celgene, Eli Lilly, Galderma, Incyte, Mayne Pharmaceuticals, MoonLake Immunotherapeutics, Novartis, Pfizer, Regeneron Pharmaceuticals Inc., Sanofi, Target Pharma, Trevi Therapeutics, and UCB; and a speaker for Amryt Pharma, Chiesi, and Krystal Biotech. Amal Assa\u2019ad has received research grants from AbbVie, Aimmune Therapeutics, Astellas, DBV Technologies, NIH, and Sanofi. Irene Lara-Corrales has received grants\/research support from AbbVie, Arcutis, Bristol Myers Squibb, Clementia, EB Medical Research Foundation, EB Research Partnership, Eli Lilly, La Roche-Posay, Mayne Pharma, Physicians Services Incorporated (PSI) Foundation, Regeneron Pharmaceuticals Inc., Sanofi, SkIN Canada, and Timber; has received honoraria or consultation fees from Abeona, Avicanna, Eli Lilly, Galderma, Incyte, Ipsen, L'Or\u00e9al, Novartis, and Sanofi; has participated in company sponsored speaker\u2019s bureau for AbbVie, Alexion, Eli Lilly, Novartis, and Sanofi; and received other support for being a board member of DEBRA Canada and Camp Libert\u00e8 and Children International Summer Villages (CISV). Dina Katabi is a co-founder of Emerald and may hold stock and\/or stock options in the company; is a board member of Cyclerion and The Marconi Society; and receives funding from Amgen, Aspen Neuroscience, BlueRock, Gwangju Institute of Science and Technology, Helmsley Charitable Trust, IBM, J&J, NIH, NSF, Rett Syndrome Research Trust, Sanofi, Takeda, The Michael J. Fox Foundation, and Verge Genomics. Hariharan Rahul and Rumen Hristov are co-founders of Emerald and may hold stock and\/or stock options in the company. Siqiao Ruan and Pranav Krishna are employees of Emerald and may hold stock and\/or stock options in the company. Karen J. Chandross and Annie Zhang are employees of Sanofi and may hold stock and\/or stock options in the company. Rajan Gupta is a former employee and consultant for Sanofi; is a current employee of Medivant Pharma and may hold stock and\/or stock options in the company. Marius Ardeleanu is an employee and shareholder of Regeneron Pharmaceuticals Inc.","order":1,"name":"Ethics","label":"Conflicts of Interest","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"The study was conducted in accordance with the Declaration of Helsinki, the International Conference on Harmonisation Good Clinical Practice guideline, and applicable regulatory requirements. An independent data and safety monitoring committee conducted blinded monitoring of patient safety data. The local institutional review board or ethics committee at each study center oversaw trial conduct and documentation. All patients, or their parents\/guardians, provided written informed consent before participating in the trial. Pediatric patients provided assent according to the Ethics Committee (Institutional Review Board [IRB]\/Independent Ethics Committee)-approved standard practice for pediatric patients at each participating center. 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