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Title: Disease’s overview Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@37374a5e Item: Title: Scientific rationale of Rimeporide repositioning in DMD Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@4671e53b Item: Title: Summary of clinical data with Rimeporide in adults Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@2db7a79b Item: Title: Materials and methods Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@6950e31 Children: Item: Title: Investigational medicinal product Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@b7dd107 Item: Title: Study design and conduct Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@42eca56e Item: Title: Selection of doses in the study Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@52f759d7 Item: Title: Study assessments Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@7cbd213e Item: Title: Sample management and analysis Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@192d3247 Item: Title: Quality assurance Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@3ecd23d9 Item: Title: Statistical methods and analysis Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@569cfc36 Children: Item: Title: Population PK analysis Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@43bd930a Item: Title: PD exploratory analysis Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@33723e30 Item: Title: Results Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@64f6106c Children: Item: Title: Disposition of subjects and demographics Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@553a3d88 Item: Title: Primary objective: safety of Rimeporide after repeated dosing in young DMD boys Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@7a30d1e6 Item: Title: Secondary objective: pharmacokinetic profile of Rimeporide in young DMD boys Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@5891e32e Item: Title: Exploratory pharmacodynamic biomarkers Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@cb0ed20 Children: Item: Title: Imaging biomarkers Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@8e24743 Item: Title: Serum biomarkers Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@74a10858 Item: Title: Discussion Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@23fe1d71 Item: Title: Conclusions Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@28ac3dc3 Item: Title: Funding Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@32eebfca Item: Title: Declaration of Competing Interest Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@4e718207 Item: Title: Acknowledgements Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@1d371b2d Item: Title: References Destination: edu.harvard.hul.ois.jhove.module.pdf.PdfSimpleObject@543c6f6d Info: Title: Rimeporide as a first- in-class NHE-1 inhibitor_ Results of a phase Ib trial in young patients with Duchenne Muscular Dystrophy Author: Stefano C. Previtali Subject: Pharmacological Research, 159 (2020) 104999. doi:10.1016/j.phrs.2020.104999 Keywords: Duchenne Muscular Dystrophy; NHE-1; Rimeporide; Safety; Pharmacokinetic; Cardiomyopathy; Creator: Elsevier CreationDate: Fri Aug 21 16:37:12 CEST 2020 ModDate: Fri Aug 21 16:37:12 CEST 2020 ID: 0x54abbb9d7556a5f99845777d26ec7b42, 0x225f1526ba66ce4d9ffcd56386d0d91f Filters: FilterPipeline: FlateDecode FilterPipeline: DCTDecode Images: Image: NisoImageMetadata: FormatName: image/jpg CompressionScheme: JPEG ImageWidth: 236 ImageHeight: 298 ColorSpace: RGB BitsPerSample: 8 BitsPerSampleUnit: integer Image: NisoImageMetadata: FormatName: image/jpg CompressionScheme: JPEG ImageWidth: 248 ImageHeight: 272 ColorSpace: RGB BitsPerSample: 8 BitsPerSampleUnit: integer Image: NisoImageMetadata: FormatName: image/png ImageWidth: 1500 ImageHeight: 715 BitsPerSample: 8 BitsPerSampleUnit: integer Filter: FlateDecode Image: NisoImageMetadata: FormatName: image/jpg CompressionScheme: JPEG ImageWidth: 1500 ImageHeight: 930 ColorSpace: RGB BitsPerSample: 8 BitsPerSampleUnit: integer Image: NisoImageMetadata: FormatName: image/jpg CompressionScheme: JPEG ImageWidth: 1058 ImageHeight: 2596 ColorSpace: RGB BitsPerSample: 8 BitsPerSampleUnit: integer Fonts: Type0: Font: BaseFont: GZMUYR+CharisSIL-Italic Encoding: Identity-H ToUnicode: true Font: BaseFont: WCOROL+CharisSIL Encoding: Identity-H ToUnicode: true Type1: Font: BaseFont: CBMPIA+AdvOT596495f2+fb FontSubset: true FirstChar: 1 LastChar: 5 FontDescriptor: FontName: CBMPIA+AdvOT596495f2+fb Flags: Symbolic FontBBox: 27, 0, 921, 878 FontFile3: true EncodingDictionary: Differences: true ToUnicode: true Font: BaseFont: CBMPKF+AdvOT7fb33346.I+20 FontSubset: true FirstChar: 146 LastChar: 146 FontDescriptor: FontName: CBMPKF+AdvOT7fb33346.I+20 Flags: Nonsymbolic FontBBox: -503, -355, 1096, 878 FontFile3: true Encoding: WinAnsiEncoding Font: BaseFont: CBMPJE+AdvOT1efcda3b.B FontSubset: true FirstChar: 40 LastChar: 121 FontDescriptor: FontName: CBMPJE+AdvOT1efcda3b.B Flags: Nonsymbolic FontBBox: -89, -257, 956, 998 FontFile3: true Encoding: WinAnsiEncoding Font: BaseFont: CBMPJC+AdvOT596495f2+20 FontSubset: true FirstChar: 2 LastChar: 150 FontDescriptor: FontName: CBMPJC+AdvOT596495f2+20 Flags: Symbolic FontBBox: -477, -355, 1103, 878 FontFile3: true EncodingDictionary: Differences: true ToUnicode: true Font: BaseFont: CBNAJB+AdvOT596495f2+22 FontSubset: true FirstChar: 1 LastChar: 138 FontDescriptor: FontName: CBNAJB+AdvOT596495f2+22 Flags: Symbolic FontBBox: 4, -257, 884, 878 FontFile3: true EncodingDictionary: Differences: true ToUnicode: true Font: BaseFont: CBMPIB+AdvOT7fb33346.I FontSubset: true FirstChar: 38 LastChar: 243 FontDescriptor: FontName: CBMPIB+AdvOT7fb33346.I Flags: Serif, Nonsymbolic FontBBox: -160, -257, 1000, 980 FontFile3: true Encoding: WinAnsiEncoding Font: BaseFont: CBMPJD+AdvOT8608a8d1+20 FontSubset: true FirstChar: 1 LastChar: 1 FontDescriptor: FontName: CBMPJD+AdvOT8608a8d1+20 Flags: Symbolic FontBBox: -970, -341, 2000, 1023 FontFile3: true EncodingDictionary: Differences: true ToUnicode: true Font: BaseFont: CBMPIC+AdvOT7fb33346.I+fb FontSubset: true FirstChar: 1 LastChar: 3 FontDescriptor: FontName: CBMPIC+AdvOT7fb33346.I+fb Flags: Symbolic FontBBox: -130, -229, 907, 878 FontFile3: true EncodingDictionary: Differences: true ToUnicode: true Font: BaseFont: CBMPFO+AdvOT596495f2 FontSubset: true FirstChar: 37 LastChar: 239 FontDescriptor: FontName: CBMPFO+AdvOT596495f2 Flags: Serif, Nonsymbolic FontBBox: -79, -257, 920, 984 FontFile3: true Encoding: WinAnsiEncoding Font: BaseFont: CBNAHB+AdvOT596495f2+03 FontSubset: true FirstChar: 1 LastChar: 3 FontDescriptor: FontName: CBNAHB+AdvOT596495f2+03 Flags: Symbolic FontBBox: -677, -533, 832, 1008 FontFile3: true EncodingDictionary: Differences: true ToUnicode: true Font: BaseFont: CBMPGP+AdvOT0de51fd2 FontSubset: true FirstChar: 46 LastChar: 121 FontDescriptor: FontName: CBMPGP+AdvOT0de51fd2 Flags: Nonsymbolic FontBBox: -13, -250, 992, 947 FontFile3: true Encoding: WinAnsiEncoding CIDFontType0: Font: BaseFont: WCOROL+CharisSIL CIDSystemInfo: Registry: Adobe Registry: Identity Supplement: 0 FontDescriptor: FontName: WCOROL+CharisSIL Flags: Serif, Symbolic FontBBox: -692, -533, 3000, 1270 FontFile3: true Font: BaseFont: GZMUYR+CharisSIL-Italic CIDSystemInfo: Registry: Adobe Registry: Identity Supplement: 0 FontDescriptor: FontName: GZMUYR+CharisSIL-Italic Flags: Serif, Symbolic, Italic FontBBox: -716, -533, 3058, 1270 FontFile3: true XMP: application/pdf doi:10.1016/j.phrs.2020.104999 Rimeporide as a first- in-class NHE-1 inhibitor_ Results of a phase Ib trial in young patients with Duchenne Muscular Dystrophy Stefano C. Previtali Teresa Gidaro Jordi Díaz-Manera Alberto Zambon Stephanie Carnesecchi Pascale Roux-Lombard Pietro Spitali Mirko Signorelli Cristina Al-Khalili Szigyarto Camilla Johansson Julian Gray Delphine Labolle Florence Porte Thomé Jacqueline Pitchforth Joana Domingos Francesco Muntoni Duchenne Muscular Dystrophy NHE-1 Rimeporide Safety Pharmacokinetic Cardiomyopathy Elsevier Pharmacological Research, 159 (2020) 104999. doi:10.1016/j.phrs.2020.104999 journal Pharmacological Research © 2020 The Authors. 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