Dear Fellow H2 Families,
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My name is Casey McPherson, founder of the To Cure a Rose (TCAR) Foundation, and now CEO of AlphaRose Therapeutics. Like you, my family’s life changed forever when my daughter Rose was diagnosed with a genetic disorder due to a mutation in the HNRNPH2 gene. Out of that journey, TCAR was born—with a mission to accelerate treatments and build better systems of drug development for children like Rose.
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Fast forward to today, we helped create RareLabs, the first lab for families doing drug development, as well as AlphaRose Therapeutics Pbc, a public benefit biotech company, with a focus on commercializing ultra rare disease treatments. Our goal is to help create a sustainable path for all ultra rare genetic conditions, so that all patients with these diseases have access to treatments.
As you may or may not have heard, we are developing an ASO (antisense oligonucleotide) treatment that we licensed to our commercial company, AlphaRose, with the goal of starting a clinical trial at some point. In order to successfully complete a trial on such a small patient population, we aim to complete a baseline study on any potential patients using technology, where every patient can be seen as their own “control.” We believe this is critical to help communicate better with regulators and insurance companies whether or not an ultra rare treatment is effective.
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I’m writing to share an exciting opportunity to participate in this groundbreaking baseline study designed to better understand HNRNPH2 and help us prepare for an upcoming potential clinical trial. This is not a clinical trial for the treatment, but every patient that is to be considered a candidate in the upcoming clinical trial will need to complete this study. This study is decentralized, meaning most activities will be done from home, reducing the burden on your family while contributing to this critical research.
Why This Study Matters
There are currently no approved treatments for HNRNPH2-related disorders, and the variability in symptoms makes it challenging to design clinical trials. This study aims to:
- Capture detailed data about daily functioning, motor skills, communication, and behavior through at-home tools (like wearable sensors and video tasks).
- Develop reliable measures to track progress in future treatment trials.
- Share data with researchers worldwide via our partnership with organizations such as Simons Searchlight to accelerate the path to therapies.
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How It Works
1. At-Home Activities:
- Your child will wear lightweight sensors on their ankles for a few weeks (removed at night).
- You’ll record short videos of simple, everyday activities (e.g., playing, communicating) using a secure app.
2. Two (2) Clinic Visits: A one-day visit to a team of doctors specializing in pediatric neurodevelopmental disorders at the beginning of the study, another visit at the end of the study.
3. Totally voluntary: You can pause or withdraw at any time.
What’s Next
If you’re interested, please visit our Study Page to enroll or contact Dr. Huiping Zhu (Email: huiping@tocurearose.org; Phone: 281-323-0073).
Every participant brings us closer to a treatment, and to a future where our children can thrive. This study is IRB-approved, and your family’s privacy is our priority. All data will be stored securely, and only de-identified data will be shared with collaborators.
A Personal Note
Rose is my inspiration, but your children are my motivation. Together, we can turn hope into action. Thank you for considering joining this effort, and for being part of a community that refuses to accept the status quo.
With gratitude,
Casey McPherson
Founder, TCAR Foundation