Sharing the Science: Update on Gene Therapy and Natural History Data

Even though we cannot meet in person in 2020, we want you to hear what has been happening in MLIV research! We will be holding a Zoom meeting for our families around the world on May 11, 2020 at 10 a.m. ET. This meeting will be a live conference (recorded for those who cannot attend) where families will learn about gene therapy and what it could mean for your children; about what has been learned from natural history clinical research; about clinical recommendations for care of people with MLIV. This live meeting will also include an open session for questions where families can ask about these topics, as well as ask questions of the doctors on any topic.

Agenda

10:00 am – Welcome, Rebecca Oberman and Randy Gold; Introduction, Dr. Sue Slaugenhaupt

10:10am – Progress on Gene Therapy: What, Why, How for MLIV Treatment, Dr. Yulia Grishchuk

10:30am – Questions for Dr. Grishchuk

10:40am – What has been Learned from Natural History Data Collection? Dr. Albert Misko

11:00am – Questions for Dr. Misko

11:10am – Rehabilitative Medicine and Care for People with MLIV, Dr. Brian Wishart

11:30am – Question for Dr. Wishart

11:40am – Open questions from families

12:00pm – Concluding remarks

Meeting Link

Time: May 11, 2020 10:00 AM Eastern Time (US and Canada)
Join Zoom Meeting: https://zoom.us/j/92442755212
Meeting ID: 924 4275 5212
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