MLD Newborn Screening Enables Access To Gene Therapy
Documentary Screening & Newborn Screening Discussion
Thursday Morning @ 7am –South Ballroom
(the South Ballroom is the Main meeting room)
Sequencing Hope follows the journey of two families, one receiving the recently FDA-approved gene therapy, and one not eligible for therapy. The peek into gene therapy is a fascinating behind the scenes look at the diagnostic and approval journey, the preparation for therapy, and you will even experience the patient receiving the therapy.
We know this screening follows a fun offsite evening with the Revvity team, but we promise to stimulate you with lots of coffee before, and good conversation after, the 43-minute film is over. Even if you roll out of bed a bit slower than normal, please join us mid-event!
The post-discussion will include a brief Q&A about gene therapy, and then we’ll dive into a short update on the MLD RUSP nomination that went into expert review this summer, and we will finish oof our time together asking & learning more about how we can help you save the lives of MLD babies in your state.
This screening of Sequencing Hope at the 2024 APHL Newborn Screening Symposium is sponsored by MLD Foundation, whose leadership has been working with families, researchers, and clinicians for 25 years … and with policy makers, newborn screening, and industry for over 15 years.
More About Sequencing Hope
Sequencing Hope
Directed by Maribeth Romslo & Lindsey Seavert
Premiered at the Minneapolis/St. Paul International Film Festival in April of 2024
Sequencing Hope follows the intimate and often gut-wrenching journey of an Alabama family grappling with the unimaginable risks and potential rewards of new frontiers in medicine as their toddler becomes the first patient in the US to receive an experimental gene therapy treatment for a rare disease.
Directors Lindsey Seavert (Love Them First) and Maribeth Romslo (Raise Your Voice) return to MSPIFF with another deeply intimate, observational portrait of families whose children are afflicted with a rare disease, and examines the high stakes of altering genes as they explore how far a family will go to save their child’s life.
https://mspfilm.org/show/sequencing-hope/