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Cited by

• 2018Alone in a Crowd? Parents of Children with Rare Diseases’ Experiences of Navigating the Healthcare System219 cited
• 2016The supportive care needs of parents with a child with a rare disease: results of an online survey168 cited
• 2010Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years148 cited
• 2019The Caregiving Experiences of Fathers and Mothers of Children With Rare Diseases in Italy: Challenges and Social Support Perceptions142 cited
• 2016Social/economic costs and health-related quality of life in patients with rare diseases in Europe121 cited
• 2015The Supportive Care Needs of Parents With a Child With a Rare Disease: A Qualitative Descriptive Study118 cited
• 2012Rare diseases, orphan drugs, and their regulation in Asia: Current status and future perspectives117 cited
• 2017Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)103 cited
• 2016Health-related quality of life and a cost-utility simulation of adults in the UK with osteogenesis imperfecta, X-linked hypophosphatemia and fibrous dysplasia85 cited
• 2010Access to orphan drugs despite poor quality of clinical evidence82 cited
• 2014Participation in society for people with a rare diagnosis56 cited
• 2023Rare diseases: why is a rapid referral to an expert center so important?48 cited