Pediatric Drug Development Workshop

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Keynote Address: A Family’s Outlook on the SMA Diagnoses of Their Daughter and Son

Session Chair(s)

Christina Bucci-Rechtweg, MD

Global Head, Maternal Health and Pediatric Regulatory Policy

Novartis , United States

Cheryl Yoder will talk about the difference in outlook their family had between the SMA diagnoses of their daughter and son.

The Yoder’s daughter was diagnosed in 2012, at a time when no treatments were on the horizon. Just three years later, in 2015, her son was able to take part in a Phase 2 clinical trial of nusinersen.

Speaker(s)

Speaker

Cheryl Yoder, RN

Cure SMA, United States

Patient Advocate

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