Fighting for life and hope
May 2026
Dear Editor
I am grandmother to an 8-year-old granddaughter diagnosed at age five with mucopolysaccharidoses (MPS1). Since she was past the age for blood transfusion, our only option has been weekly infusions of an enzyme replacement therapy. She is progressing well except that the enzyme replacement does not cross the brain barrier, and her developmental delays are apparent.
What our family advocates for is continued research funding for gene therapies that will cross the brain barrier (some good news recently for MPS2 children in that regard). My daughter rallied with the National MPS Society at the FDA (the US Food and Drug Administration) in Washington, DC, in March to prompt accelerated approvals for rare genetic disorders.
In the everyday quest of attending to the needs of a child with MPS, the new onus is fighting the Trump administration’s policies relating to healthcare, FDA, CDC (Centers for Disease Control and Prevention), Medicaid and all systems that can give life and hope to our family. We fear daily the loss of support that gives my precious granddaughter her chances for longevity.
As with all relatives of people with rare diseases, life changes in an instant. I will fight as long as possible to ensure that Trump’s disastrous policies are overthrown. I never predicted that my country would be so foreign to me.
Sincerely,
Sydney Gingrow
Greensboro, NC, USA
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