Husam Younis, PhD

2024 FUTURES Exon skipping

A brief introduction on the rationale behind exon skipping therapies, followed by an overview of current and investigative exon skipping therapies.
Key insights were shared by Dr. Angel Angelov, Dr. Ash Dugar, Lianna Orlando, PhD, Dr. Kevin M. Flanigan, Laura Torrente, PhD, Alayna Tress, MPH, Karin Lucas, PhD, Mahasweta Girgenrath, Phd, Jessica Duis, MD, Husam Younis, PharmD, PhD.

Lianna Orlando, PhD

Senior Director of Research

CureDuchenne

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Ash Dugar, PhD

Senior Vice President, Medical Affairs

Dyne Therapeutics

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Kevin Flanigan, MD

Director, Center for Gene Therapy Nationwide Children's Hospital

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Karin Lucas, Ph.D

West Medical Science Liaison Team Lead, Global Medical Affairs Sarepta Therapeutics

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Laura Torrente, PhD

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Alayna Tress, MPH

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Mahasweta Girgenrath, Phd

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Jessica Duis, MD

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Husam Younis, PhD

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Dr. Angel Angelov

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Avidity Biosciences: Presentation and discussion on initial data from EXPLORE44 Clinical Trial

This webinar, hosted by CureDuchenne and Avidity Biosciences, presents the initial data from the EXPLORE 44 clinical trial. The trial evaluates the efficacy and safety of Dalzota (AOC 1044) for treating Duchenne muscular dystrophy (DMD) in patients amenable to exon 44 skipping. Key findings include a significant increase in exon skipping and dystrophin production, with a notable reduction in creatine kinase levels, indicating potential muscle health improvements. The discussion also covers the future plans for the trial and the urgency to advance treatment options for DMD patients.

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

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Diana Castro, MD

Founder and Director of the Neurology and Neuromuscular Care Center

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Husam Younis, PhD

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Sarah Boyce

President and CEO of Avidity Biosciences

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Beginning the Journey - diagnosed 0-3 yearsSpreading Wisdom - diagnosed 6+ years