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Expert Series: Expanded Access and Compassionate Use of Experimental Therapies

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 “Expanded Access” and “Compassionate Use” are terms that are utilized in our rare disease community, but what do they mean? How do patients learn about, get involved, better understand the qualifications, and talk with their doctor about participation in either of these programs? Join MitoAction and Dr. Koenig as we dive deep into better understanding the differences and overlap between “Expanded Access” and “Compassionate Use” therapies.

compassionate use, expanded access, experimental therapies, expert series

About the Speaker

Mark Kay Koenig, MD

Mark Kay Koenig, MD

Dr. Mary Kay Koenig, a pediatric neurologist at Children’s Memorial Hermann Hospital and tenured professor in the Department of Pediatrics, Division of Child and Adolescent Neurology, at McGovern Medical School, is a member of the American Academy of Pediatrics, the American Academy of Neurology, and the Child Neurology Society. She is an officer in The Mitochondrial Medicine Society and board member of the Tuberous Sclerosis Alliance.

Dr. Koenig’s research interests are focused on clinical studies related to mitochondrial disease, neuro-metabolic disorders, tuberous sclerosis complex, and other neuro-genetic disorders.

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May 9
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Expert Series: How to Keep Airways Clear and Breathing Great – Bulbar Function and Respiratory Muscles

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May 9
12:00 pm - 1:00 pm

Expert Series: How to Keep Airways Clear and Breathing Great – Bulbar Function and Respiratory Muscles

May 15
7:00 pm - 8:00 pm

Expert Series: Serial Casting and Toe Walking

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Last Presentation

Expert Series: How to Keep Airways Clear and Breathing Great – Bulbar Function and Respiratory Muscles
Presented April 11, 2025

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