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Hunter Syndrome / MPS II - FDA Rare Disease Listening Session

Thank you for your interest in FDA's Rare Disease Patient Listening Session!

A Listening Session teleconference will be co-hosted by FDA's Patient Affairs Staff and the National Organization for Rare Disorders (NORD) during which the patients or caregivers of patients affected by Hunter Syndrome (mucopolysaccharidosis II or MPS II) will have an opportunity to share first-hand with FDA staff their experiences of living with this disease. The teleconference will be held on Tuesday, February 4, 2020 from 1:30 - 3:00 pm Eastern Time. There are a very limited number of openings for participating in the session, therefore participants will be accepted on a first-come-first-serve basis assuming the participant meets the specific requirements. Those who are selected will be notified by NORD or FDA.
1.Are you a patient or caregiver of a patient with Hunter Syndrome / MPS II?(Required.)
2.What is your name (first name and last initial only)?(Required.)
3.If you are the caregiver - What is the name of the patient (first name and last initial)?
4.In what city and state do you live?
5.Are you available and interested in participating in a teleconference call to discuss your disease experience on Tuesday, February 4, 2020 from 1:30 - 3 pm ET?(Required.)
6.Can someone from the FDA or NORD contact you?(Required.)
7.What is your email address?(Required.)
8.What is your phone number?(Required.)
9.What is the patient's gender?
10.What is the patient's age?
11.How old was the patient when they were diagnosed with this disease?(Required.)
12.Please select the symptoms that the patient has currently or previously experienced and describe below (check all that apply)(Required.)
13.At what age did the patient begin experiencing the neurological symptoms or cognitive symptoms (like mental impairment) of Hunter Syndrome? Please enter n/a if this question does not apply to you.(Required.)
14.Has the patient experienced development regression? Please describe. For example, did the patient gain and then lose a milestone such as speech, cognitive abilities, etc.?(Required.)
15.Has the patient received enzyme replacement therapy (ERT)?(Required.)
16.Has the patient received a stem cell transplant / Hematopoietic Stem Cell Transplantation (HSCT)?(Required.)
17.Please list the age the patient first received a stem cell transplant / Hematopoietic Stem Cell Transplantation (HSCT), write N/A if not applicable.(Required.)
18.Is the patient currently or has the patient ever participated in a clinical trial?(Required.)
19.If you answered YES in Q18 - How did you decide whether to enroll in a gene therapy clinical trial vs. experimental drug clinical trial?
20.Briefly describe how the patient is currently managing the disease or its symptoms. This might include, but is not limited to, ERT, medications, physical therapy, medical devices, surgery, mental/behavioral therapy, speech therapy, etc.
21.Briefly describe what you consider to be the most problematic symptom of this disease:
22.Briefly describe what you feel is the biggest burden of living with this disease:
23.What worries you the most about Hunter Syndrome/MPS II?
24.(Optional) Is there anything else you would like to share about your experience with this disease?
25.(Optional) Do you have any comments, questions, or concerns about the Listening Session?
26.(Optional) How did you hear about the Listening Session?
27.If you are affiliated with a patient organization, please indicate which organization here: