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Expanding Horizons: Launching a Rare Disease Nonprofit Amid Government Funding Challenges
Geri Landman, MD, MPH, cofounder and chair of Moonshots for Unicorns, talks about how the lack of government funding for PGAP3 research led her to take it upon herself to work toward a cure for her daughter.
In this installment of the "Expanding Horizons" series, Geri Landman, MD, MPH, cofounder and chair of Moonshots for Unicorns, pediatrician at Palo Alto Foundation Medical Group, addresses the challenges of insufficient government and pharmaceutical funding for rare diseases, and how Moonshots for Unicorns came to be as a response to those challenges.
Driven by the urgent need for treatments, especially for conditions like PGAP3, the nonprofit organization seeks to pioneer a pathway for single-gene conditions, with the ultimate goal of making research more accessible and affordable for families. Landman also advocates for a decentralized approach to government-sponsored research, proposing a model where funds directly empower existing patient organizations to accelerate the pace of finding cures for rare diseases.
According to Landman, "If government rare disease funding wants to be more useful to rare disease families, it has to pick up the pace."
Check out the first part of the "Expanding Horizons" feature series here. To learn more about Moonshots for Unicorns, visit www.moonshotsforunicorns.org
Missed Opportunities for Salpingectomy Highlight Gaps in Ovarian Cancer Prevention
August 27th 2025Many patients later diagnosed with high-grade serous cancer had prior surgical or genetic testing opportunities that could have reduced their ovarian cancer risk, underscoring the need to expand opportunistic salpingectomy and preventive strategies.
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Infertility Coverage Boosts ART Use and Pregnancy Success: Richard A. Brook, MS, MBA
August 26th 2025In this episode, Richard A. Brook, MS, MBA, discusses his study showing that infertility treatment coverage increases assisted reproductive technology (ART) use and improves pregnancy outcomes.
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Interhospital Transfers Occur Less Frequently for Uninsured Patients
August 27th 2025Patients with acute respiratory failure who’ve been placed on a mechanical ventilator are less likely to be transferred to high-volume centers if they are uninsured, thus increasing their odds of mortality.
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Hope on the Horizon for Underserved Patients With Multiple Myeloma: Joseph Mikhael, MD
August 12th 2025Explore the disparities in multiple myeloma treatment and how new initiatives aim to improve clinical trial participation among underrepresented patients during a conversation with Joseph Mikhael, MD, MEd, FRCPC, FACP, FASCO, chief medical officer of the International Myeloma Foundation.
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Global Burden of MASH-Related Liver Cancer Rising Sharply Among Older Adults
August 26th 2025The global incidence, deaths, and disability-adjusted life years tied to metabolic dysfunction–associated steatohepatitis (MASH)-related liver cancer have more than doubled in older adults since 1990, with a heavy impact on low- and middle-income countries.
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