THURSDAY, JUNE 1
4:00 – 7:00 pm Conference Registration
5:00 – 6:00 pm Mentor Program Gathering
5:30 – 7:00 pm Happy Hour
Social time with a pizza station, snacks and drinks
7:00 – 9:00 pm Getting to Know You
Make new friends and get to know each other during this fun, interactive kick-off session for the whole family. Be sure to check your “Getting to Know You Card” provided at registration. It contains you and your family’s scheduled time at each of our four stations, with a different activity every half hour.
FRIDAY, JUNE 2
9:00 – 9:15 am Welcome
Patricia Wood
President, NBIA Disorders Association
9:15 – 10:15 am Keynote Address
Put Good Out
Life’s daily distractions and disappointments can sometimes feel overwhelming, but that doesn’t mean we should just surrender. Diane Kolvek discovered the healing power in doing one good deed each day after she lost her sister to a random act of violence. In the process, her outlook, her life and her world changed for the better. She is working on her next book, Ruling the World Through Good, and shares her methods and insights with us.
Diane Kolvek
Actor, Inspirational Speaker
Vice President, Ameritas
Omaha, Nebraska
10:15 – 10:30 am Coffee Break
10:30 – 11:30 am Clinical Trials: Issues to Consider
Drs. Hogarth and Vichinsky share their experience and insights on clinical trials. They explain the process and describe the issues that those contemplating joining any trial should consider.
Penelope Hogarth, MD
Associate Professor, Department of Neurology
Oregon Health & Science University
Elliott Vichinsky, MD
Director of Hematology/Oncology
UCSF Benioff Children’s Hospital Oakland
Professor of Pediatrics at the University of California, San Francisco
11:30 – 12:30 pm Breakout Sessions for PKAN/BPAN/MPAN/PLAN
These family-only sessions are being led by parent facilitators. This is your chance to speak openly to others living with or caring for someone with the same disorder. We ask that those with other affiliations, such as researchers and industry representatives, not attend these sessions.
12:30 – 1:30 pm Lunch
Hotel Atrium
1:30 – 2:00 pm Natural History Studies in NBIA: Challenges and New Approaches
What natural history studies are and why they are important for studying ultra-rare disorders like NBIA. This will be the foundation for discussing the OHSU team’s new natural history study designs and how they address some of the challenges of NBIA research.
Allison Gregory, MS, Certified Genetic Counselor
Genetic Counselor & Assistant Professor, Department of Molecular and Medical Genetics
Oregon Health & Science University
2:00 – 2:30 pm Discovering Strength in Numbers
Meg Talley Dyer speaks via a taped conversation on what she has learned about being an advocate since 2014, the year her daughter was diagnosed with BPAN. Talley Dyer has discovered there is strength in numbers when she teamed up with the NBIA Disorders Association. Wood discusses a key source of that strength—the grant awards program aimed at uncovering therapies and, ultimately, a cure for NBIA.
Meg Talley Dyer
Board Chair, NBIA Disorders Association
Patricia Wood
President, NBIA Disorders Association
2:30 – 3:00 pm The Power of Giving and Social Media
Join in celebrating our fundraising volunteers whose efforts bring attention, support and resources to the fight for NBIA treatments and cures. Drop by our Hospitality Suite to learn more about fundraising and social media right after this session, or from 7-8 p.m. Saturday (before the Dessert Social starts). Contact our Development Director at info@NBIAdisorders.org or 760-402-5686.
Melissa Woods
Social Media Director, NBIA Disorders Association
3:00 – 3:15 pm Coffee Break
3:15 – 4:15 pm Breakout Sessions: Each hour-long session covers a different topic, giving participants an opportunity to follow their specific interests. See descriptions below.
3:15 – 4:15 pm The Pace of Progress
Do you sometimes feel as if the path to treatment is too slow? Dr. Hogarth will reprise her talk from our 2015 conference with an overview – in a very visual way – of the pace of research discovery. Although the speed of discovery may feel slow to some, it’s been accelerating for all NBIA disorders.
Penelope Hogarth, MD
Associate Professor, Department of Neurology
Oregon Health & Science University
3:15 – 4:15 pm Coping with an NBIA diagnosis
Deb Wojcik, a licensed clinical social worker, discusses the trauma of serious medical illness and coping strategies for families caring for a loved one with a disorder like NBIA. As a private practitioner and member of the clinical staff at the Cancer Wellness Center, she has spent more than 25 years helping people cope with life-limiting medical illness, grief, loss and life transitions.
Deb Wojcik
Cancer Wellness Center
Northbrook, Illinois
3:15 – 4:15 pm Advocating for Your Child
Lawyer and educator Michael Kiser, who has represented students for over 15 years, shares tips and strategies to help you more effectively advocate for your child at school. You know your child’s strengths and needs, and you can help identify and push for the resources your child needs to succeed. Learn about the special education process, how to negotiate with the school and school district, and when to consider hiring a qualified professional to assist you.
Michael Kiser
Attorney
Chicago, Illinois
3:15 – 4:15 pm What’s the Scoop on Fundraising and Social Media?
Does the thought of tweeting, posting and “liking” online make your head spin? Do you keep meaning to set up a FirstGiving page but don’t know where to start?
4:15 – 4:45 pm Meet the Board
In the past year, the NBIA Board of Trustees has added five new faces. Hear a little bit about each trustee, new and old, and don’t hesitate to ask questions or share your ideas.
4:45 – 5:00 pm NBIA Around the World
Learn more about the NBIA Alliance, an international collaboration of NBIA organizations, and the work on NBIA disorders being accomplished around the world.
Patricia Wood
President, NBIA Disorders Association
5:00 – 5:30 pm TIRCON Deferiprone Clinical Trial Update
Dr. Vichinsky discusses the deferiprone clinical trial and what happens next.
Elliott Vichinsky, MD
Director of Hematology/Oncology at UCSF Benioff Children’s Hospital Oakland
Professor of Pediatrics at the University of California, San Francisco
SATURDAY, JUNE 3
8:30 - 8:45 am Announcements
8:45 – 9:10 am Research Update
Dr. Jackowski will report on her progress on a PKAN mouse model and on PKAN therapeutics.
Suzanne Jackowski, PhD
Member, St. Jude Children’s Research Hospital Faculty
Memphis
9:10 – 9:35 am Research Update
Dr. Hogarth discusses progress in BPAN research.
Penelope Hogarth, MD
Associate Professor, Department of Neurology
Oregon Health & Science University
9:35 – 10:00 am Research Update
Dr. Kotzbauer shares information on his studies of the PLA2G6 enzyme and the current approach for developing therapies for INAD.
Paul Kotzbauer, MD, PhD
Associate Professor, Department of Neurology
Washington University School of Medicine
St. Louis
10:00 – 10:15 am Coffee Break
10:15 – 11:00 am Research Update
Dr. Hayflick provides updates on a broad range of research projects, including those funded by the NBIA Disorders Association and others around the world.
Susan Hayflick, MD
Chair, Department of Molecular and Medical Genetics
Professor, Molecular and Medical Genetics, Pediatrics, and Neurology
Oregon Health & Science University
11:00 – 12:15 am Breakout Sessions: In separate sessions, our NBIA expert researchers answer questions from NBIA families about the status of research and therapies for each specified NBIA disorder.
11:00 – 11:30 am BPAN (Dr. Penelope Hogarth) & MPAN (Dr. Susan Hayflick)
11:45 – 12:15 pm PKAN (Dr. Susan Hayflick & Dr. Suzanne Jackowski) & PLAN (Dr. Paul Kotzbauer)
1:00 – 2:15 pm Lunch at Lake County Forest Preserve’s Half Day Shelter A & B in Vernon Hills
The Knights of Columbus Councils of Deerfield and Vernon Hills are generously cooking and catering a picnic lunch at the shelter. It is a 12-minute drive from the hotel, and directions are available for those driving.
2:15 – 2:30 pm Group Photo
If you have a Team NBIA shirt, please wear it for our traditional group photo.
2:30 – 2:45 pm Memorial Tribute
We remember our NBIA friends and loved ones in this brief ceremony.
2:45 – 3:15 pm Rare Bear Pickup and Group Photo with Bears Rare Bears for NBIA individuals
Harmony 4 Hope Care Packages Harmony 4 Hope Care Packages for all those 18 and younger will be distributed by this non-profit organization.
3:15 – 4:45 pm Team NBIA videos and pictures
4:45 – 5:00 pm Bus leaves to return to hotel
8:00 – 11:00 pm Dessert Social
Silent auction and drawing for gift baskets donated by NBIA families, friends and board members, with all proceeds going to NBIA Disorders Association. Dancing with Grand DJ Entertainment
SUNDAY, JUNE 4
8:45 – 10:00 am Breakout Session for Adult NBIA Individuals (over 18)
Dr. Byer, a licensed clinical psychologist and coordinator of the Young Adult Program at the Cancer Wellness Center, facilitates a group session for NBIA adults. In his job, Byer holds support groups, provides counseling and supervises therapists-in-training. This session will explore challenges, coping skills and other topics of interest to NBIA-affected adults attending the conference.
Evan Byer, PhD
Clinical Psychologist
Cancer Wellness Center
Chicago
9:15 – 10:15 am Will Gene Editing Fix NBIA?
A new gene-editing technology, CRISPR/Cas, is being touted for its potential to revolutionize medicine. It is showing promise as a treatment for certain cancers. Rare diseases are more complicated, but research already is underway in several labs using CRISPR technology for Duchenne muscular dystrophy. Eugene Wyatt, a post-doctoral fellow researching CRISPR/Cas and muscular dystrophy, will discuss his work and the potential for other rare disorders in a Q & A format with Mary Ann Roser. Wyatt works in a genetics lab at Northwestern University’s Feinberg School of Medicine.
Eugene Wyatt, PhD
Postdoctoral Fellow
Center for Genetic Medicine at Northwestern University’s Feinberg School of Medicine
Chicago
Mary Ann Roser
Board Member, NBIA Disorders Association
10:15 – 11:15 am Recreation & Leisure Activities
Recreation and leisure play important roles in the health and well-being of individuals with disabilities and their families. Play allows children to understand and relate to the world as they interact with their siblings, parents and community. Learn from NBIA parent Matthew Ritzman and recreation experts Tracey Hoffman and Ahren Crawford how to adapt games, promote playing and explore recreational ideas to maximize functioning and promote a higher quality of life.
Tracey Crawford
Executive Director of the Northwest Special Recreation Association
Rolling Meadows, Illinois
Ahren Hoffman
Certified Therapeutic Recreation Specialist
Director, American Specialty Toy Retailing Association
Chicago
Matthew Ritzman
Board Member, NBIA Disorders Association
11:15 – 11:30 am Conference Evaluations
11:30 – 12:00 pm Closing Ceremony
Join us as we share our experiences of the conference, and view highlights in a video centering on our theme of “Team NBIA.”
