Externally-Led Patient Focused Drug Development (EL-PFDD)

Voice of the Patient Report

The comments collected may be used in the final PFDD Voice of the Patient report (with identifying information removed).

The Wiskott-Aldrich Foundation is holding an Externally-Led Patient Focused Drug Development (EL-PFDD) meeting on Wiskott-Aldrich Syndrome (WAS) and X-Linked Thrombocytopenia (XLT). This virtual meeting is an important opportunity for patients and caregivers to inform FDA representatives, academic and scientific researchers, medical professionals, and pharmaceutical companies about personal experiences regarding the symptoms and daily impact of WAS/XLT, as well as thoughts on current and future approaches to therapies. 


About PFDD Meetings

Learn more about the PFDD 

Meeting Objectives

Watch January 12, 2023 WAF EL-PFDD Community Webinar

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