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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
Despite Record ACA Enrollment, Report Reveals Underinsured Americans are in Crisis
November 21st 2024Despite significant progress in expanding health insurance coverage since the Affordable Care Act (ACA) was enacted, millions of Americans still face critical gaps in access and affordability to health care.
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New Study Finds Risk Groups, Outpatient Care Barriers in Chronic Liver Disease
November 20th 2024Patients with chronic liver disease who were unable to establish care were 85% more likely to require recurrent hospitalizations. This group included a disproportionate number of women and individuals with physical limitations affecting their health.
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Exploring Racial, Ethnic Disparities in Cancer Care Prior Authorization Decisions
October 24th 2024On this episode of Managed Care Cast, we're talking with the author of a study published in the October 2024 issue of The American Journal of Managed Care® that explored prior authorization decisions in cancer care by race and ethnicity for commercially insured patients.
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OS Better With Belantamab Mafodotin Triplet vs Daratumumab in R/R MM
November 19th 2024The key secondary end point of overall survival (OS) was met in the DREAMM-7 trial of belantamab mafodotin (Blenrep; GSK) for the treatment of patients with relapsed/refractory multiple myeloma (R/R MM).
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