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How social pharmaceutical innovations are addressing problems of availability, accessibility and affordability of drugs for rare diseases.
Risdiplam utilization, adherence, and associated health care costs for patients with spinal muscular atrophy: a United States retrospective claims database analysis.
Validation of Noninvasive Assessment of Pulmonary Gas Exchange in Patients with Chronic Obstructive Pulmonary Disease during Initial Exposure to High Altitude.
Allelic prevalence and geographic distribution of cerebrotendinous xanthomatosis.
Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS).