Experience with Edasalonexent Demonstrates Ability of 4 to ... Poster FINAL.pdf · Experience with...

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Experience with Edasalonexent Demonstrates Ability of 4 to 7 Year-old Boys with Duchenne Muscular Dystrophy to Take Softgel Capsules in Clinical Trials Maria Mancini MHP 1 , Gigi Shafai PharmD 1 , Joanne Donovan MD PhD 1 , Sachin Chandran PhD 1 , Richard Finkel MD 2 1 Catabasis Pharmaceuticals, Boston, MA; 2 Nemours Children’s Health System, Orlando, FL Background Methods Study Design Results — Capsule Swallowing Ability at Screening Results — Capsule Selection by Age and Geography Results — Capsule Selection by Age and Geography Conclusion Results — Capsule Size and Swallowing Ability Acknowledgements Questions? [email protected] Presented at the 2020 PPMD Annual Conference ‣ Patients and families ‣ Patient groups ‣ PolarisDMD Phase 3 Site Staff ‣ MoveDMD Phase 2 Site Staff ‣ Catabasis team ‣ Thanks to PPMD and MDA for generous grant support for patient travel in the MoveDMD Phase 2 trial

Transcript of Experience with Edasalonexent Demonstrates Ability of 4 to ... Poster FINAL.pdf · Experience with...

Page 1: Experience with Edasalonexent Demonstrates Ability of 4 to ... Poster FINAL.pdf · Experience with Edasalonexent Demonstrates Ability of 4 to 7 Year-old Boys with Duchenne Muscular

Experience with Edasalonexent Demonstrates Ability of 4 to 7 Year-old Boys with Duchenne Muscular Dystrophy to Take Softgel Capsules in Clinical TrialsMaria Mancini MHP1, Gigi Shafai PharmD1, Joanne Donovan MD PhD1, Sachin Chandran PhD1, Richard Finkel MD2

1Catabasis Pharmaceuticals, Boston, MA; 2Nemours Children’s Health System, Orlando, FL

Background

Methods

Study Design

Results — Capsule Swallowing Ability at Screening Results — Capsule Selection by Age and Geography

Results — Capsule Selection by Age and Geography Conclusion

Results — Capsule Size and Swallowing Ability

Acknowledgements

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

‣ Patients and families

‣ Patient groups

‣ PolarisDMD Phase 3 Site Staff

‣ MoveDMD Phase 2 Site Staff

‣ Catabasis team

‣ Thanks to PPMD and MDA for generous grant support for patient travel in the MoveDMD Phase 2 trial

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