September 26, 2025
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To Our Community,
This year, I had the profound honor of joining the Cure Rare Disease (CRD) team as Chief Advancement Officer. Every day, I stand alongside families and patients who have heard the most devastating words a doctor can deliver, and still refuse to surrender. Families who reject the idea that “nothing can be done.” Families who refuse to believe that their child’s life is too rare to matter.
I know what it means to stand in that place. I know because I’ve stood there myself.
In 2017, my son, Nash, was diagnosed with SMARD1, a fatal disease so rare there wasn’t a single published research paper on it. He was just 11 months old, with a life expectancy of 13 months. In that moment, my world shattered; and then, piece by piece, I rebuilt myself into someone who would not accept that my son’s story was already written. Just like the families who find their way to CRD, I became a mother unwilling to concede to inevitability.
In the wake of Nash’s diagnosis, we founded SmashSMARD with nothing but desperation, determination, and the unshakeable love of a family fighting for one child’s life. We had no scientific training. No roadmap. No promises. Just grit and the belief that impossible is not a verdict.
And in three years, we did what had never been done: we developed the first, and only gene therapy for SMARD1.
The barriers we broke.
The battles we fought.
The countless nights spent navigating research, manufacturing, regulation, logistics, and hope. Stretched thin, but never breaking.
It was all worth it. Because that therapy gave us two more precious years with Nash. Two more birthdays. Two more summers filled with laughter, music, and miracles we were told would never come. Nash may have lost his battle on March 18, 2024; but he won the war.
Since joining CRD, I often think about how different our journey could have been if we hadn’t had to build that path from scratch.
Maybe I could have held my son instead of shouldering the entire burden of creating a treatment.
Maybe my four boys could have had a childhood shaped by wonder instead of survival.
That is what Cure Rare Disease exists to do. We lift that crushing weight off families. We transform isolation into collaboration, despair into direction, and heartbreak into breakthroughs that were once unimaginable.
CRD has ignited partnerships across science, academia, manufacturing, regulatory, and legal landscapes to create a pathway where none existed. We take the fractured, complex world of drug development and forge a unified, customizable way to deliver treatments to patients once deemed “too rare to treat.”
But here’s the thing: Science doesn’t move on its own. It moves when people do. Behind every breakthrough is a community that refuses to look away.
As a member of the Cure Rare Disease community, your support has enabled breakthroughs for each of the diseases we are working to treat.
In 2025 alone, we received Orphan Drug Designation from the FDA for our LGMD2i gene therapy and SCA3 antisense oligonucleotide (ASO). We developed next-generation, liver-detargeting AAV vectors to deliver CRISPR-based gene therapies for rare mutations of Duchenne muscular dystrophy. Each of these advancements brings us closer to moving these therapies into clinical trials.
As we close this year and look ahead to 2026, I invite you to stand with us once again. Every discovery, every regulatory milestone, every patient whose story we have the chance to change, all of it begins with the compassion and commitment of people like you.
Your generosity powers science, fuels hope, and reminds every family facing the unthinkable that they are not alone.
Please consider making a year-end donation to Cure Rare Disease so that, together, we can continue pushing the boundaries of what’s possible for those living with ultra-rare diseases.
Your gift directly accelerates the development of life-changing therapies and brings us closer to clinical trials that once seemed out of reach. Join us in building a future where no family fights alone, and every patient has a fighting chance.
Your support today is the difference between “someday” and “now.”
With gratitude,
Brittany Stineman
Chief Advancement Officer, Cure Rare Disease
Cure Rare Disease, a non-profit organization (Tax ID number 82-2473513), is qualified as a tax-exempt organization under section 501(c)(3) of the IRS and has been designated as a “public charity” under section 170.