ARCHIVED: Ohio Rare Disease Advisory Council (RDAC) Meeting 5/5/2022

Ohio Rare Disease Advisory Council (RDAC)

5/5/2022 1 - 3 p.m.

Virtual Teams Meeting

Agenda

Adjourn

Minutes

A quorum is established.

Guests: Charlene York, Krista Kano, Emily (no last name), Christina Dew, Andrew Henry, Joseph Ewig, Nancy M., Matt Forney, Vincent Squillance, S. Crapes, Sara Barkley, RLwchd, Natalie Hicks, Laura Wallrabensteirn, Steve Lonsinger, Rose Leonhard, Megan Richwine, Jack Brubaker, Libby Brunsvold, Courtney Saunders, Glenn Wirth, Tamara Hess, MaryEllen Baker, Ashley Ruth, Brett Logan, Kate Segal, Kimberly Mathews, ODH Parent Consultant (ODH Admin Support)

The minutes were shared and a few corrections were noted:

• December West asked that her child's diagnoses be changed to (2).
• Randi Clites asked for the "wish list items."

With these two corrections, Annie Ross Womack made motion to approve. Senator Craig seconded. Vote taken and approved.

Andrea Hoffman provided the RDAC a presentation regarding cystic fibrosis. Randi thanked Andrea for the presentation. Dr. Kopp added there may be those not diagnosed or underdiagnosed in minority and varying ethnic groups. This could potentially double national numbers. Access to treatments depends upon the type of CF genetic mutation.

December West provide the RDAC a presentation regarding Alport Syndrome. Randi thanked December for the presentation.

Kelly Maynard provide the RDAC a presentation regarding Duchenne syndrome. Randi thanked Kelly for the presentation.

Questions:

Dr. Ahuja - Are you aware of gene therapy for Duchenne? Kelly's foundation funded the pre-clinical work that occurred at Nationwide Children's Hospital.

There will be three families sharing at the next RDAC meeting. She also asked the RDAC members to let her know if you would like to share at the next meeting as to what you are doing around research, diagnosis, and treatment (5-7 minutes).

Patrick Londergan shared in the report it would be good to include information as to:

• The current state of rare diseases.
• How could the legislature support the work occurring in the rare disease community.
• Make the report informative and helpful to provide education to decision makers.
• What can be gleaned from the report that will make a difference for individuals.

Randi Clites led a discussion regarding the 3 pillars - Research, Diagnosis, and Treatment.

There was also an in-depth conversation and learning opportunity regarding the ODH Children with Medical Handicaps program (CMH). Patrick Londergan answered many questions and share information about several of the program components. In the chat the CMH website link was shared.

Randi Clites led a discussion regarding the subcommittee co-chairs. Co-chairs will be as follows:

• Diagnosis - Eileen Sullivan/Dr. Manickam.
• Research - Dr. Pena/Dr. Kopp.
• Treatment - Kelly Maynard/Tiffany Sammons.

Randi will work with Kim Mathews to send out information for RDAC members to join the subcommittees.

Next steps:

• Virtual meetings can occur through June 30, 2022.

Next meeting dates:

• June 23, 2022 (1 - 3 p.m.) - virtual.
• September 22, 2022 (2 - 4 p.m.) - in person.
• December 15, 2022 (2 - 4 p.m.) - in person.

Senator Craig made a motion to adjourn. Patrick Londergan seconded. Meeting adjourned.