
OpenAlex is a bibliographic catalogue of scientific papers, authors and institutions accessible in open access mode, named after the Library of Alexandria. It's citation coverage is excellent and I hope you will find utility in this listing of citing articles!
If you click the article title, you'll navigate to the article, as listed in CrossRef. If you click the Open Access links, you'll navigate to the "best Open Access location". Clicking the citation count will open this listing for that article. Lastly at the bottom of the page, you'll find basic pagination options.
Requested Article:
Patient reported outcome measures in rare diseases: a narrative review
Anita Slade, Fatima Isa, Derek Kyte, et al.
Orphanet Journal of Rare Diseases (2018) Vol. 13, Iss. 1
Open Access | Times Cited: 171
Anita Slade, Fatima Isa, Derek Kyte, et al.
Orphanet Journal of Rare Diseases (2018) Vol. 13, Iss. 1
Open Access | Times Cited: 171
Showing 1-25 of 171 citing articles:
Therapies for rare diseases: therapeutic modalities, progress and challenges ahead
Erik Tambuyzer, Benjamin Vandendriessche, Christopher P. Austin, et al.
Nature Reviews Drug Discovery (2019) Vol. 19, Iss. 2, pp. 93-111
Open Access | Times Cited: 270
Erik Tambuyzer, Benjamin Vandendriessche, Christopher P. Austin, et al.
Nature Reviews Drug Discovery (2019) Vol. 19, Iss. 2, pp. 93-111
Open Access | Times Cited: 270
Castleman disease
Antonino Carbone, Margaret Borok, Blossom Damania, et al.
Nature Reviews Disease Primers (2021) Vol. 7, Iss. 1
Open Access | Times Cited: 122
Antonino Carbone, Margaret Borok, Blossom Damania, et al.
Nature Reviews Disease Primers (2021) Vol. 7, Iss. 1
Open Access | Times Cited: 122
Retinitis Pigmentosa: Current Clinical Management and Emerging Therapies
Xuan‐Thanh‐An Nguyen, Lude Moekotte, Astrid S. Plomp, et al.
International Journal of Molecular Sciences (2023) Vol. 24, Iss. 8, pp. 7481-7481
Open Access | Times Cited: 57
Xuan‐Thanh‐An Nguyen, Lude Moekotte, Astrid S. Plomp, et al.
International Journal of Molecular Sciences (2023) Vol. 24, Iss. 8, pp. 7481-7481
Open Access | Times Cited: 57
<p>The use of patient-reported outcome research in modern ophthalmology: impact on clinical trials and routine clinical practice</p>
Tasanee Braithwaite, Melanie Calvert, Alastair Gray, et al.
Patient Related Outcome Measures (2019) Vol. Volume 10, pp. 9-24
Open Access | Times Cited: 109
Tasanee Braithwaite, Melanie Calvert, Alastair Gray, et al.
Patient Related Outcome Measures (2019) Vol. Volume 10, pp. 9-24
Open Access | Times Cited: 109
Establishing Patient Registries for Rare Diseases: Rationale and Challenges
Vanessa Boulanger, Marissa Schlemmer, S. Rossov, et al.
Pharmaceutical Medicine (2020) Vol. 34, Iss. 3, pp. 185-190
Open Access | Times Cited: 98
Vanessa Boulanger, Marissa Schlemmer, S. Rossov, et al.
Pharmaceutical Medicine (2020) Vol. 34, Iss. 3, pp. 185-190
Open Access | Times Cited: 98
The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey
David Dingli, Joana E. Matos, Kerri Lehrhaupt, et al.
Annals of Hematology (2022) Vol. 101, Iss. 2, pp. 251-263
Open Access | Times Cited: 43
David Dingli, Joana E. Matos, Kerri Lehrhaupt, et al.
Annals of Hematology (2022) Vol. 101, Iss. 2, pp. 251-263
Open Access | Times Cited: 43
Socio-economic costs of rare diseases and the risk of financial hardship: a cross-sectional study
Claudia Ching Yan Chung, Nicole Y.T. Ng, Yvette Nga Chung Ng, et al.
The Lancet Regional Health - Western Pacific (2023) Vol. 34, pp. 100711-100711
Open Access | Times Cited: 23
Claudia Ching Yan Chung, Nicole Y.T. Ng, Yvette Nga Chung Ng, et al.
The Lancet Regional Health - Western Pacific (2023) Vol. 34, pp. 100711-100711
Open Access | Times Cited: 23
The Use of Patient-Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment
Amanda Whittal, Michela Meregaglia, Elena Nicod
Patient (2021) Vol. 14, Iss. 5, pp. 485-503
Open Access | Times Cited: 54
Amanda Whittal, Michela Meregaglia, Elena Nicod
Patient (2021) Vol. 14, Iss. 5, pp. 485-503
Open Access | Times Cited: 54
Systematic Review of N-of-1 Studies in Rare Genetic Neurodevelopmental Disorders
Annelieke R. Müller, Marion M. Brands, Peter M. van de Ven, et al.
Neurology (2021) Vol. 96, Iss. 11, pp. 529-540
Open Access | Times Cited: 53
Annelieke R. Müller, Marion M. Brands, Peter M. van de Ven, et al.
Neurology (2021) Vol. 96, Iss. 11, pp. 529-540
Open Access | Times Cited: 53
European Reference Networks: challenges and opportunities
Birutė Tumienė, Holm Graeßner, Irene M.J. Mathijssen, et al.
Journal of Community Genetics (2021) Vol. 12, Iss. 2, pp. 217-229
Open Access | Times Cited: 44
Birutė Tumienė, Holm Graeßner, Irene M.J. Mathijssen, et al.
Journal of Community Genetics (2021) Vol. 12, Iss. 2, pp. 217-229
Open Access | Times Cited: 44
Quality of life, physical functioning, and psychosocial function among patients with achondroplasia:a targeted literature review
Constantinos Constantinides, Sarah Landis, James Jarrett, et al.
Disability and Rehabilitation (2021) Vol. 44, Iss. 21, pp. 6166-6178
Open Access | Times Cited: 34
Constantinos Constantinides, Sarah Landis, James Jarrett, et al.
Disability and Rehabilitation (2021) Vol. 44, Iss. 21, pp. 6166-6178
Open Access | Times Cited: 34
What role can decentralized trial designs play to improve rare disease studies?
J. Moore, Noah Goodson, Paul Wicks, et al.
Orphanet Journal of Rare Diseases (2022) Vol. 17, Iss. 1
Open Access | Times Cited: 25
J. Moore, Noah Goodson, Paul Wicks, et al.
Orphanet Journal of Rare Diseases (2022) Vol. 17, Iss. 1
Open Access | Times Cited: 25
N‐of‐1 trials in epilepsy: A systematic review and lessons paving the way forward
Victoria Defelippe, Eva H. Brilstra, Willem M. Otte, et al.
Epilepsia (2024) Vol. 65, Iss. 11, pp. 3119-3137
Open Access | Times Cited: 5
Victoria Defelippe, Eva H. Brilstra, Willem M. Otte, et al.
Epilepsia (2024) Vol. 65, Iss. 11, pp. 3119-3137
Open Access | Times Cited: 5
The sixth international RASopathies symposium: Precision medicine—From promise to practice
Karen W. Gripp, Lisa Schill, Lisa Schoyer, et al.
American Journal of Medical Genetics Part A (2019) Vol. 182, Iss. 3, pp. 597-606
Open Access | Times Cited: 38
Karen W. Gripp, Lisa Schill, Lisa Schoyer, et al.
American Journal of Medical Genetics Part A (2019) Vol. 182, Iss. 3, pp. 597-606
Open Access | Times Cited: 38
Patient and clinician opinions of patient reported outcome measures (PROMs) in the management of patients with rare diseases: a qualitative study
Olalekan Lee Aiyegbusi, Fatima Isa, Derek Kyte, et al.
Health and Quality of Life Outcomes (2020) Vol. 18, Iss. 1
Open Access | Times Cited: 38
Olalekan Lee Aiyegbusi, Fatima Isa, Derek Kyte, et al.
Health and Quality of Life Outcomes (2020) Vol. 18, Iss. 1
Open Access | Times Cited: 38
Psychological Aspects of Congenital Hypogonadotropic Hypogonadism
Andrew Dwyer, Neil Smith, Richard Quinton
Frontiers in Endocrinology (2019) Vol. 10
Open Access | Times Cited: 37
Andrew Dwyer, Neil Smith, Richard Quinton
Frontiers in Endocrinology (2019) Vol. 10
Open Access | Times Cited: 37
Parental health spillover effects of paediatric rare genetic conditions
You Wu, Hareth Al‐Janabi, Andrew Mallett, et al.
Quality of Life Research (2020) Vol. 29, Iss. 9, pp. 2445-2454
Closed Access | Times Cited: 36
You Wu, Hareth Al‐Janabi, Andrew Mallett, et al.
Quality of Life Research (2020) Vol. 29, Iss. 9, pp. 2445-2454
Closed Access | Times Cited: 36
A Psychometric Evaluation of the Motor-Behavioral Assessment Scale for Use as an Outcome Measure in Rett Syndrome Clinical Trials
Melissa Raspa, Carla Bann, Angela Gwaltney, et al.
American Journal on Intellectual and Developmental Disabilities (2020) Vol. 125, Iss. 6, pp. 493-509
Open Access | Times Cited: 36
Melissa Raspa, Carla Bann, Angela Gwaltney, et al.
American Journal on Intellectual and Developmental Disabilities (2020) Vol. 125, Iss. 6, pp. 493-509
Open Access | Times Cited: 36
Patient-reported outcomes and patient-reported outcome measures in interstitial lung disease: where to go from here?
Meena Kalluri, Fabrizio Luppi, Ada Vancheri, et al.
European Respiratory Review (2021) Vol. 30, Iss. 160, pp. 210026-210026
Open Access | Times Cited: 28
Meena Kalluri, Fabrizio Luppi, Ada Vancheri, et al.
European Respiratory Review (2021) Vol. 30, Iss. 160, pp. 210026-210026
Open Access | Times Cited: 28
Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi)
Daphne H. Schoenmakers, Shanice Beerepoot, Sibren van den Berg, et al.
Orphanet Journal of Rare Diseases (2022) Vol. 17, Iss. 1
Open Access | Times Cited: 19
Daphne H. Schoenmakers, Shanice Beerepoot, Sibren van den Berg, et al.
Orphanet Journal of Rare Diseases (2022) Vol. 17, Iss. 1
Open Access | Times Cited: 19
Toward responsible clinical n-of-1 strategies for rare diseases
Victoria Defelippe, Ghislaine J. M. W. van Thiel, Willem M. Otte, et al.
Drug Discovery Today (2023) Vol. 28, Iss. 10, pp. 103688-103688
Open Access | Times Cited: 13
Victoria Defelippe, Ghislaine J. M. W. van Thiel, Willem M. Otte, et al.
Drug Discovery Today (2023) Vol. 28, Iss. 10, pp. 103688-103688
Open Access | Times Cited: 13
Approaches to reduce the uncertainty in the clinical efficacy of orphan drugs
V. V. Оmelyanovskiy, Sergey I. Kutsev, P. A. Mukhortova, et al.
Kachestvennaya Klinicheskaya Praktika = Good Clinical Practice (2025), Iss. 4, pp. 82-96
Open Access
V. V. Оmelyanovskiy, Sergey I. Kutsev, P. A. Mukhortova, et al.
Kachestvennaya Klinicheskaya Praktika = Good Clinical Practice (2025), Iss. 4, pp. 82-96
Open Access
Health-Related Quality of Life (HRQoL) Assessments in Research on Patients with Adult Rare Solid Cancers: A State-of-the-Art Review
Catarina S. Padilla, Cristiane Decat Bergerot, Kim Dijke, et al.
Cancers (2025) Vol. 17, Iss. 3, pp. 387-387
Open Access
Catarina S. Padilla, Cristiane Decat Bergerot, Kim Dijke, et al.
Cancers (2025) Vol. 17, Iss. 3, pp. 387-387
Open Access
Treatments and therapies for symptoms and clinical manifestations of adult type 1 myotonic dystrophy: A scoping review
A. Childs, Robert D. Henderson, Daniel James Henderson, et al.
Journal of the Neurological Sciences (2025) Vol. 472, pp. 123470-123470
Open Access
A. Childs, Robert D. Henderson, Daniel James Henderson, et al.
Journal of the Neurological Sciences (2025) Vol. 472, pp. 123470-123470
Open Access
Development and evaluation of RhizoQOL, a quality-of-life caregiver-reported survey for rhizomelic chondrodysplasia punctata, a rare peroxisomal disorder
Mousumi Bose, Tahra C. Anglade, Chelsea I. Donlon, et al.
Orphanet Journal of Rare Diseases (2025) Vol. 20, Iss. 1
Open Access
Mousumi Bose, Tahra C. Anglade, Chelsea I. Donlon, et al.
Orphanet Journal of Rare Diseases (2025) Vol. 20, Iss. 1
Open Access