Online survey:

    *   Ultragenyx Pharmaceutical is currently conducting a survey
         to understand how LC-FAOD impacts people’s lives. We invite
         patients and caregivers to take the survey at:                                    www.FAODsurvey.com

Children’s Hospital of Pittsburgh/UPMC - Medical Genetics - One Children's Hospital Drive
Faculty Pavilion 1st Floor - 4401 Penn Avenue - Pittsburgh, PA 15224
​Office:  412.692.5099
 Keith McIntire -  Coordinator, INFORM - keith.mcintire@chp.edu

International Network for Fatty Acid Oxidation Research and Management

Patient Advocacy Summit:

  •    The individual must be living with or caring for someone living with LC-FAOD of any age
​         who is articulate and comfortable with participating in group discussions. 

  •    Participants who have experience with the following LC-FAOD conditions would be
​          ideal for this meeting:

        *   Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency

        *   Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency

        *   Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency

        *   Trifunctional Protein (TFP) Deficiency

        *   Carnitine-acylcarnitine translocase (CACT) deficiency

        *   We would like to hear your Thoughts and feelings related to your LC-FAOD journey
            and areas of unmet need within the community.  This will help us better understand
            the needs of the LC-FAOD patient community and gain a deeper understanding  
            of how best to communicate and work with the community. The meeting will also
            provide participants an opportunity to offer feedback about programs being
            developed for the LC-FAOD community.  There will be a few representatives from                     Ultragenyx at the meeting. This Patient Advocacy Summit is not related to                               participation in any clinical trial.   

  •    Only minimal information will be shared with Ultragenyx from those people 
        who express interest in moving forward with attending the meeting and agree to allow             their information to be shared. Participants must be well enough and available to travel
        safely to Nashville, Tennessee on May 20, 2017. All travel will be arranged and travel
        expenses covered for participants by Ultragenyx.    

  •    Contact Anna Cunningham at Snow Companies at:
     acunningham@snow-companies.com or 757-707-5683.

  
       Please note that participation is limited and not all interested candidates may
    be able to participate.

Ultragenyx Pharmaceutical Inc. has 3 current opportunities for FAOD patients and caregivers to provide feedback about their experiences. 

  INFORM Families

Telephone research study:

      *   The purpose of the study is to explore the patient and caregiver experience of FAOD.
           The research study is being conducted by a company called Adelphi Values on behalf
           of Ultragenyx.

      *   As part of the study, Adelphi Values is conducting 60 minute interviews with patients
          and caregivers about their experience with LC-FAOD. This interview will be conducted
          via telephone or Skype at a time convenient for the patient.

      *   All information provided during the study will be treated confidentially and will 
          be kept anonymous.

      *   Participation is optional. If your patients or their caregivers would like to take 
           part or if they would like any further information about the study, please have 
           them contact Bryony Brookes at Adelphi Values at:                                            bryony.brookes@adelphivalues.com

​       Telephone: 617-720-0001 / Fax: +44 (0)1625-577-328