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Caregiver-perceived behavioral challenges in fragile X syndrome and implications for measuring treatment benefit in clinical trials.

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Title: Caregiver-perceived behavioral challenges in fragile X syndrome and implications for measuring treatment benefit in clinical trials.
Authors: Merikle E; Harmony Biosciences, Plymouth Meeting, PA, USA. emerikle@harmonybiosciences.com., Tich N; Harmony Biosciences, Plymouth Meeting, PA, USA., Sebree T; 1323 Consulting, LLC, Johnson City, TX, USA., Hagerman R; Medical Investigation of Neurodevelopmental Disorders (MIND) Institute, University of California Davis Medical Center, Sacramento, CA, USA.; Department of Pediatrics, University of California Davis School of Medicine, Sacramento, CA, USA., O'Quinn S; Perissos Inc, Wake Forest, NC, USA., Nomikos G; Harmony Biosciences, Plymouth Meeting, PA, USA., Bzdek K; Harmony Biosciences, Plymouth Meeting, PA, USA.
Source: Journal of patient-reported outcomes [J Patient Rep Outcomes] 2026 Apr 01; Vol. 10 (1). Date of Electronic Publication: 2026 Apr 01.
Publication Type: Journal Article
Journal Info: Publisher: Springer International Publishing Country of Publication: Germany NLM ID: 101722688 Publication Model: Electronic Cited Medium: Internet ISSN: 2509-8020 (Electronic) Linking ISSN: 25098020 NLM ISO Abbreviation: J Patient Rep Outcomes Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: <searchLink fieldCode="AU" term="%22Merikle+E%22">Merikle E</searchLink>; Harmony Biosciences, Plymouth Meeting, PA, USA. emerikle@harmonybiosciences.com.<br /><searchLink fieldCode="AU" term="%22Tich+N%22">Tich N</searchLink>; Harmony Biosciences, Plymouth Meeting, PA, USA.<br /><searchLink fieldCode="AU" term="%22Sebree+T%22">Sebree T</searchLink>; 1323 Consulting, LLC, Johnson City, TX, USA.<br /><searchLink fieldCode="AU" term="%22Hagerman+R%22">Hagerman R</searchLink>; Medical Investigation of Neurodevelopmental Disorders (MIND) Institute, University of California Davis Medical Center, Sacramento, CA, USA.; Department of Pediatrics, University of California Davis School of Medicine, Sacramento, CA, USA.<br /><searchLink fieldCode="AU" term="%22O'Quinn+S%22">O'Quinn S</searchLink>; Perissos Inc, Wake Forest, NC, USA.<br /><searchLink fieldCode="AU" term="%22Nomikos+G%22">Nomikos G</searchLink>; Harmony Biosciences, Plymouth Meeting, PA, USA.<br /><searchLink fieldCode="AU" term="%22Bzdek+K%22">Bzdek K</searchLink>; Harmony Biosciences, Plymouth Meeting, PA, USA.
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  Data: <searchLink fieldCode="JN" term="%22101722688%22">Journal of patient-reported outcomes</searchLink> [J Patient Rep Outcomes] 2026 Apr 01; Vol. 10 (1). <i>Date of Electronic Publication: </i>2026 Apr 01.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Springer+International+Publishing%22">Springer International Publishing </searchLink><i>Country of Publication: </i>Germany <i>NLM ID: </i>101722688 <i>Publication Model: </i>Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>2509-8020 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2225098020%22">25098020 </searchLink><i>NLM ISO Abbreviation: </i>J Patient Rep Outcomes <i>Subsets: </i>MEDLINE
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        Value: 10.1186/s41687-026-01054-9
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      – Code: eng
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              Text: 2026 Apr 01
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