Deborah Horwitz & Paul Nierman
Our grandson is now in fourth grade, learning remotely due to the pandemic, as are some other students in his grade. He misses his friends terribly, but says that at least his recess is a lot more fun. While the kids in school have to stand in one spot and stretch and move a little, he shoots a few baskets on his driveway or works on complex jigsaw puzzles. His activities are all remote e.g. children’s musical theater, piano lessons, art classes, and although soccer and Little League were cancelled in the spring, he is playing intrasquad fall soccer. He still takes over 25 treatments each day (multiple enzymes, antibiotics, inhaled nebulizer treatments, breathing treatments & airway clearance therapies, basic nutrition through a port in his tummy).
It is imperative to find treatments for infection control and the immune system; excess inflammation; mucus clearance from the lungs; gastrointestinal complications including more effective delivery of enzymes to replace the non-functioning pancreas, and other affected systems. More than half of the work at the CF Foundation Therapeutics Lab is concentrated on nonsense and other rare mutations, including an initiative to create a cell culture bank with cells from people with CF who have rare mutations.
The CF Foundation is also pushing the frontiers of science on many fronts on its $500 million Path to a Cure initiative started in October 2019. As part of this initiative, the CF Foundation is pursuing new approaches to restoring CFTR activity in individuals like our grandson who have nonsense or other rare mutations that do not respond to other modulators. Some of these approaches would help everyone with CF, regardless of their mutation.
All of this takes time – lots of time. We are committed to giving until it’s done.
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