Comment from Kyrsten Lawless
AnonymousSupportAdvocacy
Summary: Kyrsten Lawless, a parent and advocate for a child with alpha-mannosidosis, supports the use of the Plausible Mechanism Framework to develop individualized gene therapies for rare genetic conditions. She argues that current enzyme replacement therapies are insufficient and costly, and urges the FDA to provide enhanced incentives and streamlined pathways for curative, one-time gene therapies that can cross the blood-brain barrier.
See attached file(s)
As a parent, I want my child to have a viable future. Investing in gene therapies for children with rare LSD's is going to be expensive, but in the long run you are setting children up to actually have a future beyond being institutionalized later in life. A one time treatment in much more cost effective in the long run when you factor in the lifetime of care of a patient, and the dependency on caregivers, and symptom management for life.
My daughter wants to be independent, and has a lot of behaviors because she doesn't want to be helped all the time.
Parents are exhausted because we are spread so thin, and it is not easy for parents with rare disease to always know how to take action and who to talk to. Primarily because no one in my immediate life truly understands or dives deep into pediatric rare disease especially LSD's because it is so depressing.
Please make a future for these children a priority, as parents feel like their life is out of control due to these devastating genetic progressive disorders, and we need hope. Why is Medicaid paying a crazy amount of money on treatment that has no end over the course of her lifetime, has extreme side effects (for her), has a very short half life (once a week), and doesn't even help her brain.
Let me know how I can help. Please contact me via email (on pdf) for questions/ concerns.