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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
Managed Care Cast Presents: BTK Inhibitors in Treatment-Naive Patients With CLL and MCL
December 26th 2024A trio of experts discuss the treatment of chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL) with Bruton tyrosine kinase (BTK) inhibitors, including cost considerations.
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HS Treatment Goals: Better Quality of Life, Not Just Control
January 3rd 2025For part 3 of our discussion with Chris Sayed, MD, we tackle several important topics in the hidradenitis suppurative (HS) and inflammatory disease space: patient quality of life, medication and treatment goals, and the possibility of a cure.
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