edasalonexent treatment in young boys with duchenne ... poster final.pdf · activation of nf-kb in...

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Edasalonexent Treatment in Young Boys with Duchenne Muscular Dystrophy Is Associated with Age-Normative Growth and Normal Adrenal Function Erika L. Finanger, MD 1 ; Richard Finkel, MD 2 ; Krista Vandenborne, PT, PhD 3 ; H Lee Sweeney, PhD 3 ; Gihan Tennekoon, MBBS, MRCS, LCRP 4 ; Perry Shieh, MD, PhD 5 ; Sabrina W. Yum, MD 4 ; Maria Mancini, MHP 6 ; James MacDougall, PhD 6 ; Joanne Donovan, MD, PhD 6 1 Oregon Health Sciences University, Portland, OR; 2 Nemours Children’s Health System, Orlando, FL; 3 University of Florida Health, Gainesville, FL; 4 The Children's Hospital of Philadelphia, Philadelphia, PA; 5 University of California, Los Angeles, Los Angeles, CA; 6 Catabasis Pharmaceuticals, Boston, MA Background Study Design Results Conclusion Results Questions? [email protected] Presented at the 2020 PPMD Annual Conference Acknowledgements ‣ Patients and families ‣ Patient groups ‣ ImagingDMD staff ‣ Site staff ‣ Catabasis team ‣ Thanks to PPMD and MDA for generous grant support for patient travel

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Page 1: Edasalonexent Treatment in Young Boys with Duchenne ... Poster FINAL.pdf · Activation of NF-KB in Duchenne Muscular Dystrophy Is a Key Factor in Disease Progression No Dystrophin

Edasalonexent Treatment in Young Boys with Duchenne Muscular Dystrophy Is Associated with Age-Normative Growth and Normal Adrenal FunctionErika L. Finanger, MD1; Richard Finkel, MD2; Krista Vandenborne, PT, PhD3; H Lee Sweeney, PhD3; Gihan Tennekoon, MBBS, MRCS, LCRP4; Perry Shieh, MD, PhD5; Sabrina W. Yum, MD4; Maria Mancini, MHP6; James MacDougall, PhD6; Joanne Donovan, MD, PhD6

1Oregon Health Sciences University, Portland, OR; 2Nemours Children’s Health System, Orlando, FL; 3University of Florida Health, Gainesville, FL; 4The Children's Hospital of Philadelphia, Philadelphia, PA; 5University of California, Los Angeles, Los Angeles, CA; 6Catabasis Pharmaceuticals, Boston, MA

Background Study Design

Results Conclusion

Results

Questions? [email protected] at the 2020 PPMD Annual Conference

Acknowledgements

‣ Patients and families

‣ Patient groups

‣ ImagingDMD staff

‣ Site staff

‣ Catabasis team

‣ Thanks to PPMD and MDA for generous grant support for patient travel

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MUSCLE DISEASE Gene editing restores dystrophin expression in … · 2020-04-13 · MUSCLE DISEASE Gene editing restores dystrophin expression in a canine model of Duchenne muscular

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Engineered DNA plasmid reduces immunity to …...Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy

Increased dystrophin production with golodirsen in ... · treated patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Methods Part 1 was a randomized, double-blind,

Review Article Understanding the Process of Fibrosis in ...Duchenne muscular dystrophy (DMD), caused by loss of dystrophin gene function. In DMD, skeletal muscle degenerates and is

Analysis of the dystrophin interactome

The Roles of the Dystrophin-Associated Glycoprotein ... · Abstract Duchenne muscular dystrophy is caused by muta- ... DGC roles in the nervous system, gained from studies in both

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Diagnosi precoce e clinica delle malattie neuromuscolari: implicazioni per la ... · 2020-04-24 · Distrofia Muscolare di Duchenne Dystrophin: The protein product of the duchenne

CARRIER DETECTION OF DUCHENNE AND BECKER MUSCULAR ... · dystrophin immunohistochemistry (Table 6b). (3) Dystrophin immunohistochemistry was more sensitive than EMG and muscle biopsy

Myocarditis and dilated cardiomyopathy...cardiomyopathies Patient, male, 34 years old Cardiac biopsies: EBV↑ + inflammation↑ CD45b (T-lymphocytes) Dystrophin-1 Dystrophin-2 Dystrophin-3