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Cracking the code: a head-to-head comparison of expert clinicians and artificial intelligence in diagnosing rare diseases.
The economic impact of caregiving for individuals with Angelman syndrome in the United States: results from a caregiver survey.
Efficacy of tranilast in preventing exacerbating cardiac function and death from heart failure in muscular dystrophy patients with advanced-stage heart failure: a single-arm, open-label, multicenter study.
Growth hormone treatment improves final height in children with X-linked hypophosphatemia.
Tranilast for advanced heart failure in patients with muscular dystrophy: a single-arm, open-label, multicenter study.