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Rare Disease Patients Face Hurdles Beyond Diagnosis, Treatment
The quest to save Grace, a patient with a rare genetic disorder, highlights systemic barriers in clinical trial access and regulatory pathways for orphan diseases.
AATD: Aiming for AAT Restoration and Clinically Meaningful Endpoints
Advancements in Alpha-1 Antitrypsin Deficiency (AATD) focus on restoring AAT levels and achieving clinically meaningful endpoints.
