PRESENTATION TITLE
Rare Disease Day Special Webinar: From Mila to Millions

MAIN PRESENTER

• BIO
  • In December 2016, Julia founded Mila's Miracle Foundation upon learning her seemingly healthy six-year-old daughter, Mila had Batten disease. Julia’s collaboration with Dr. Timothy Yu from Boston Children’s Hospital led to the first-ever drug tailored to just one person. Unfortunately, the treatment did not come soon enough for Mila. Julia is now on a mission to turn the work that went into that drug into a treatment pathway for millions of children across rare diseases.
  • Julia regularly presents at scientific meetings and conferences globally. She co-founded the N=1 Collaborative which serves as the international scientific hub for medicines, as well as EveryONE Medicines, a biotech aiming to prove a viable business model where there currently is not one. Through Mila's Miracle Foundation, Julia has raised millions of dollars to fund novel treatments and programs for children with rare genetic conditions. Additionally, Julia co-runs the first-ever single cell atlas of pediatric disease with Boston Children’s Hospital, funds basic science research in the US and Europe, and hosts meetings with industry experts and families in the rare disease community.

• BIO
  • Ms. Barnes is the Executive Director of the MN Rare Disease Advisory Council, a Minnesota state agency which was created in 2019 following grassroots efforts by a cross-sector coalition of advocates. She has over a decade of advocacy and non-profit leadership experience, having co-founded with her husband Chloe’s Fight Rare Disease Foundation in honor of her late daughter. Ms. Barnes has a number of committee roles related to rare diseases. She chairs the NIH funded Global Leukodystrophy Initiative Clinical Trials Network patient advocacy consortium, is a member of the Sickle Cell Data Collection (SCDC) Program and is a coalition leader of CureMLD.

• BIO
  • Margot A. Cousin, Ph.D., is an Assistant Professor of Medical Genetics in the Dept. of Molecular Medicine, Director of the N-or-1 Therapeutics Program in the Center for Individualized Medicine, and a KL2 Scholar through the Center for Clinical and Translational Science at Mayo Clinic. Dr. Cousin completed her PhD in Clinical and Translational Science from Mayo Clinic Graduate School of Biomedical Science in 2015. She completed her postdoctoral training in the Translational Omics Program within the Center for Individualized Medicine at Mayo Clinic. Her current research focuses on developing and translating novel therapies such as antisense oligonucleotides for rare genetic disorders.

LEARNING OBJECTIVES:

1. Identify the obstacles and challenges within our current ecosystem that have prevented the rapid advancement of treatments and therapies for children affected by rare genetic conditions.
2. Summarize the potential impact and advantages of the individualized medicines approach.
3. Describe the ways in which family advocates and experts in the rare disease community are collaborating to pave a new regulatory path for individualized medicines and expedite access to n-of-1 therapies.

ATTENDANCE / CREDIT
Text the session code (provided only at the session) to 507-200-3010 within 48 hours of the live presentation to record attendance. All learners are encouraged to text attendance regardless of credit needs. This number is only used for receiving text messages related to tracking attendance. Additional tasks to obtain credit may be required based on the specific activity requirements and will be announced accordingly. Swiping your badge will not provide credit; that process is only applicable to meet GME requirements for Residents & Fellows.

TRANSCRIPT
Any credit or attendance awarded from this session will appear on your Transcript.

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