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Sessions and Speakers

The 2019 LCA Family Conference will provide information about advances in research, deepen your understanding of the roles various organizations play in developing treatments, and provide insight into how an active patient community can support and accelerate treatment.

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9:00 – 9:15 am
Welcoming Remarks, Sofia Sees Hope
We'll kick off the day by letting you know what we've been up to, how we're supporting the LCA and rare IRD community, and how to get the most out of your conference.

9:15 – 10:00 am
Foundation Fighting Blindness
Hear what our partners at the Foundation Fighting Blindness have been up to - from their approach to tackling tough problems to fight blindness, to exciting research updates for retinal disease.
  • Ben Yerxa, CEO Foundation Fighting Blindness

10:15 - 11:00 am
The Long and Winding Road of the Human Genome Project and Gene Therapy, Dr. Katherine High

11:00 - 11:45 am
Your Voice Matters!
Hear from the US Food & Drug Administration, industry representatives, and rare disease organizations how patients and caregivers can make a real difference in moving treatments forward.  Moderated by Annette Tonti, Executive Director of Sofia Sees Hope. 
  • Jamie Ring, Head, Patient Advocacy at Spark Therapeutics, Inc.
  • Jill Dolgin, Head of Patient Advocacy at AGTC
  • Emily Milligan, Executive Director at the Barth Syndrome Foundation
  • Terri Booker, Advocate for Sickle Cell Anemia

1:00 - 2:00 pm
One Disease, Many Approaches
From gene augmentation, to gene editing, to oligonucleaotidetherapy, there’s more than one way to approach genetic therapy! The experts will walk you through these very different approaches to genetic treatment.  Moderated by Brian Mansfield, Executive Vice President Research, interim Chief Scientific Officer at Foundation Fighting Blindness.

  • Jean Bennett, MD, PhD Director, Center for Advanced Retinal and Ocular Therapeutics (CAROT), University of Pennsylvania Perelman School of Medicine; Professor, Ophthalmology and Cell & Developmental Biology
  • Tomas Aleman, MD, University of Pennsylvania Perelman School of Medicine
  • Mike Schwartz, M.S., MBA, ProQR
  • Pam Stetkiewicz, VP Program Management, Editas Medicine

2:00 - 3:00 pm
All About Clinical Trials
What’s involved in getting a treatment approved for clinical trial? What’s it like to be in a trial? How will I know if it’s safe, or if it’s the right thing to do for me and my family? In this session, you’ll hear from experts about the safeguards in place for your protection, and from patients who have been in a trial and will share their personal experiences.  Moderated by Ben Shaberman, Senior Director, Scientific Outreach & Community Engagement at Foundation Fighting Blindness.

  • Dr. Wiley Chambers, Supervisory Medical Officer in the Office of New Drugs at the FDA
  • Tami & Michael Moorehouse, Phase 1 RPE65 Trial Subject LCA/RPE65 genetic therapy trial
  • Michel Michaelides, MD, Head of Clinical Ophthalmology, MeiraGTX

3:15 - 4:00 pm
Living with an IRD Breakout Groups
We’ll set up these sessions based on interest. When you register, let us know which sessions are most interesting to you, or if there’s another topic you would like to hear about!
  • Living with an IRD: The Early Years
  • Living with an IRD: The Teen Years
  • Living with an IRD: The Adult Years
  • Genetic testing & counseling
  • Empowering your school-aged child through self-advocacy
  • Connecting Online: The Good, The Bad, The OMG I’m Blocking You
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