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This pressure does two things
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None had a placebo control group
One area thats been of particular interest is employer coverage
Furthermore, limited diagnostic capabilities, difficulty in identifying eligible patients, and variability in disease progression collectively contribute to low enrollment rates, prolonged study timelines, and compromised data integrity in rare disease trials, ultimately resulting in higher failure rates compared to non-rare disease trials 5,6
For the first time, Medicare beneficiaries may access these medications at $245/month with a $50 copay cap, starting mid-to-late 2026
