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Rarer dementias, limited options, and unaddressed needs

Commentary on “A Mixed Methods Evaluation of a Programme Exploring Pre-death Grief and Loss for Carers of People with Rarer Dementias,” by Stevens-Neck et al.

Published online by Cambridge University Press:  04 May 2023

Jeffrey Cummings*
Affiliation:
Chambers-Grundy Center for Transformative Neuroscience, Las Vegas, NV, USA Department of Brain Health, School of Integrated Health Sciences, Las Vegas, NV, USA University of Nevada Las Vegas, Las Vegas, NV, USA
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Abstract

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Type
Commentary
Copyright
© The Author(s), 2023. Published by Cambridge University Press on behalf of International Psychogeriatric Association

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References

Adachi, T. et al. (2023). Enhancing equitable access to rare disease diagnosis and treatment around the world: a review of evidence, policies, and challenges. International Journal of Environmental Research and Public Health, 20, 4732.Google Scholar
Brooks, P. J., Yang, N. N. and Austin, C. P. (2016). Gene therapy: the view from NCATS. Human Gene Therapy, 27, 713.Google Scholar
Carlin, B. P. and Nollevaux, F. (2022). Bayesian Complex Innovative Trial Designs (CIDs) and their use in drug development for rare disease. The Journal of Clinical Pharmacology, 62, S56S71.Google Scholar
Cummings, J. (2021). The role of neuropsychiatric symptoms in research diagnostic criteria for neurodegenerative diseases. The American Journal of Geriatric Psychiatry, 29, 375383.Google Scholar
Finch, N., et al. (2009). Plasma progranulin levels predict progranulin mutation status in frontotemporal dementia patients and asymptomatic family members. Brain, 132, 583591.Google Scholar
Food and Drug Administration (2023). Advancing Health Through Innovation: New Drug Therapy Approvals 2022. Silver Spring, MD: Food and Drug Administration.Google Scholar
Haendel, M. et al. (2020). How many rare diseases are there? Nature Reviews Drug Discovery, 19, 7778.Google Scholar
Hendriks, S. et al. (2021). Global prevalence of young-onset dementia: a systematic review and meta-analysis. JAMA Neurology, 78, 10801090.Google Scholar
Hoglinger, G. U. et al. (2021). Safety and efficacy of tilavonemab in progressive supranuclear palsy: a phase 2, randomised, placebo-controlled trial. The Lancet Neurology, 20, 182192.Google Scholar
Mellerio, J. E. (2022). The challenges of clinical trials in rare diseases. British Journal of Dermatology, 187, 453454.Google Scholar
Mintun, M. A. et al. (2021). Donanemab in early Alzheimer’s disease. New England Journal of Medicine, 384, 16911704.Google Scholar
Morgan, P. et al. (2018). Impact of a five-dimensional framework on R&D productivity at AstraZeneca. Nature Reviews Drug Discovery, 17, 167181.Google Scholar
Murillo-Cuesta, S. et al. (2020). The value of mouse models of rare diseases: a Spanish experience. Frontiers in Genetics, 11, 583932.Google Scholar
NORD (2022). BRIGHTER TOMORROW. A Year of Innovation, Advancement and Progress for Rare Disease. Washington, DC: NORD.Google Scholar
Panza, F. et al. (2020). Development of disease-modifying drugs for frontotemporal dementia spectrum disorders. Nature Reviews Neurology, 16, 213228.Google Scholar
Stevens-Neck, R. et al. (2023). A mixed methods evaluation of a programme exploring pre-death grief and loss for carers of people with rarer dementias. International Psychogeriatrics, In Press.Google Scholar
Tabrizi, S. J. et al. (2019). Targeting Huntingtin expression in patients with Huntington’s disease. New England Journal of Medicine, 380, 23072316.Google Scholar
Tsang, V. W. L. et al. (2019). Role of patients and parents in pediatric drug development. Therapeutic Innovation & Regulatory Science, 53, 601608.Google Scholar
van Dyck, C. H. et al. (2022). Lecanemab in early Alzheimer’s disease. New England Journal of Medicine, 388, 921.Google Scholar
Yates, N. and Hinkel, J. (2022). The economics of moonshots: value in rare disease drug development. Clinical and Translational Science, 15, 809812.Google Scholar