PCers Needed to Answer 4 Questions May 3, 2023 by Pachyonychia 2023 Voice of the Patient Update We’ve detected that Javascript is not enabled. It is required for an optimal survey taking experience. Please check your browser’s settings and make sure Javascript is turned on. Learn how to enable Javascript. Skip survey header 2023 Voice of the Patient Update The EL-PFDD meeting with FDA in 2018 was a success and has been very critical in PC drug development. Because it has been five years, we plan to send an update to FDA officials to let them know we still need treatments for PC. Some of these questions may look familiar but it is important for you to complete them again for this update to the official Voice of the Patient Report. Do not include your experience with recent clinical trials treatments in your answers. Contact Information First Name Last Name Email What is your age or the age of the person you care for? 0-2 years old 3-12 years old 13-18 years old 18-25 years old 26-45 years old 45-65 years old older than 65 In which PC gene is your specific mutation found? K6a K6b K6c K16 K17 1. Overall, how would you characterize your PC disease severity or the severity of the person you care for compared to someone without PC? MILD (While I have symptoms, I am able to, on most days, manage those symptoms and live a healthy, normal life without limitations) MODERATE (PC has an impact on my everyday life, and on some days limits my ability to function normally due to ambulation challenges, pain, etc.) SEVERE (PC is debilitating to my everyday life, and in many cases limits my abilities to live a normal life due to ambulation challenges, pain, etc.) UNBEARABLE (PC at times causes me to withdraw from society; I feel it is difficult to continue on and occasionally I have suicidal thoughts) 2. Which PC conditions have impacted your life? Check all that apply. Thickened nails Painful calluses/blisters on the soles of your feet Painful calluses/blisters on hands Painful blood vessels/nerves in calluses Deep persistent itch in feet Infections in nails or feet Painful cysts Follicular hyperkeratosis (little bumps on waist, legs, arms, etc. that cause irritation) Leukokeratosis (white growth on tongue) Trouble feeding as a baby Sharp ear pain at first bite or drink that lasts about 20-30 seconds Other – Write In Please enter an ‘other’ value for this selection. 3. How satisfied are you with the treatments available today for your PC? HIGHLY SATISFIED: Current treatments allow me to live a life with no limitations SATISFIED: Current treatments allow me to live a life with only a few limitations UNSATISFIED: Even with current treatments, I am living a life with many limitations VERY UNSATISFIED: Current treatments are unsatisfactory, and do little to help overcome the everyday disease burden 4. Have you ever utilized a mobility assistance device (e.g., wheelchair, cane, scooter, walker) or employed an alternative form of mobility (e.g. such as bikes, strollers, crawling, holding onto walls or rails, piggy-backing, holding onto another person, etc.) because of your PC? Yes, at some point during every day Yes, not every day, but at least once or more during the week Yes, on some occasions, but not every week No, never
