Preventative treatment of tuberous sclerosis complex with sirolimus: Phase I safety and efficacy results.

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Title: Preventative treatment of tuberous sclerosis complex with sirolimus: Phase I safety and efficacy results.
Authors: Capal JK; Department of Neurology, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA., Ritter DM; Division of Neurology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Franz DN; Division of Neurology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Griffith M; Division of Neurology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Currans K; Division of Behavioral Medicine and Clinical Psychology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Kent B; Division of Speech-Language Pathology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Bebin EM; Department of Neurology, University of Alabama at Birmingham, Birmingham, AL, USA., Northrup H; Division of Medical Genetics, Department of Pediatrics, McGovern Medical School at the University of Texas Health Science Center at Houston (UTHealth) and Children's Memorial Hermann Hospital, Houston, TX, USA., Koenig MK; Division of Neurology, Department of Pediatrics, McGovern Medical School at the University of Texas Health Science Center at Houston (UTHealth) and Children's Memorial Hermann Hospital, Houston, TX, USA., Mizuno T; Division of Translational and Clinical Pharmacology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Vinks AA; Division of Translational and Clinical Pharmacology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Galandi SL; Division of Pathology and Laboratory Medicine, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Zhang W; Division of Pathology and Laboratory Medicine, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Setchell KDR; Division of Pathology and Laboratory Medicine, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Kremer KM; Division of Neurology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Prada CM; Division of Genetics, Genomics, and Metabolism, Department of Pediatrics, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL, USA., Greiner HM; Division of Neurology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Holland-Bouley K; Division of Neurology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Horn PS; Division of Neurology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Krueger DA; Division of Neurology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Source: Annals of the Child Neurology Society [Ann Child Neurol Soc] 2024 Jun; Vol. 2 (2), pp. 106-119. Date of Electronic Publication: 2024 Apr 22.
Publication Type: Journal Article
Journal Info: Publisher: Wiley Periodicals LLC Country of Publication: United States NLM ID: 9918523088806676 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2831-3267 (Electronic) Linking ISSN: 28313267 NLM ISO Abbreviation: Ann Child Neurol Soc Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2831-3267
DOI:10.1002/cns3.20070