EL-PFDD Meeting on CCDS

ACD hosted an Externally-Led Patient Focused Drug Development (EL-PFDD) meeting on Cerebral Creatine Deficiency Syndromes (CCDS) on January 24, 2023. This meeting provided 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 CCDS, as well as thoughts on current and future approaches to therapies.

The CCDS Voice of the Patient Report is an impactful summary of this meeting with many quotes from caregivers and live polling results presented in an insightful manner. We invite you to read this report and learn more about our community. 

  • The PFDD Program was created by the FDA several years ago as a way to systematically gather information from patients and caregivers about their conditions, especially symptoms and daily impact, as well as thoughts on current and future approaches to therapies. This information helps inform FDA’s drug development decision making process.
  • Externally-Led PFDD’s are hosted by an organization other than the FDA, in this case the ACD.
  • EL-PFDDs give patients and caregivers a platform to share what it means to live with, or be a caregiver for someone who has one of the three CCDS, including symptoms, daily impact, and overall quality of life.
  • Discussions were focused on existing treatments for AGAT and GAMT and thoughts on future approaches to treatments for CTD
  • A CCDS “Voice of the Patient” report will be published spring 2023.
  • View the Community Orientation Webinar, held prior to the EL-PFDD Meeting here.

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