Our approach to rare disease research is grounded in our experience as patients and caregivers in clinical trials.
Our experiences in and frustrations with clinical trials in Duchenne muscular dystrophy led us to found Casimir in 2016 with one simple goal: to develop outcome measures and trials that better capture the nuances of disease progression and treatment benefit. By identifying what matters to patient populations, we can better understand the meaningfulness of an intervention.
Prior to founding Casimir, Christine McSherry spent 15 years as the executive director of the Jett Foundation, which she founded in 2001 after her son, Jett, was diagnosed with Duchenne. She has raised over $15 million for Duchenne research and patient programs. Christine has advocated for the patient perspective to be considered by the FDA when reviewing safe and potentially effective treatments for all rare diseases. In April of 2016, Christine presented the Jett Foundation's Patient Centered Outcomes report at the FDA's meeting of the Peripheral and Central Nervous System Drugs Advisory Committee.
Following her experiences participating in a half dozen clinical trials, Mindy Leffler designed a PRO program for her son, Aidan, a 14-year-old with Duchenne Muscular Dystrophy. That PRO program proved instrumental in the approval of EXONDYS51, the first FDA-approved treatment for Duchenne.
Today, Casimir works with sponsors in over 20 rare diseases in every facet of clinical services from preclinical development to Phase IV and siteless RWE studies. At every step of the process, every decision we make is grounded in an understanding of and respect for the role patient communities play in informing and participating in clinical research.
To learn more about Casimir's work in rare disease including novel outcome measure development, virtual trials and clinical services, visit out services page.
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