The Government's role in regulating, coordinating, and standardizing the response to Alzheimer's disease: Anticipated international cooperation in the area of intractable and rare diseases

Tang, Qi; Song, Peipei; Xu, Lingzhong

doi:10.5582/irdr.2016.01037

Cited by 6 publications

(3 citation statements)

References 18 publications

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“…Alzheimer's disease (AD) is the primary dementia‐causing disease and approximately 46.8 million people worldwide had dementia in 2015 . It is estimated that 5–7% of older adults who are 60 years and over will be affected by AD . The most common early symptom in AD is difficulty in remembering recently learned information.…”

Section: The Amyloid Precursor Protein (App) and The Production Of Aβmentioning

confidence: 99%

Plaque formation and the intraneuronal accumulation of β‐amyloid in Alzheimer's disease

Takahashi

Nagao

Gouras

2017

Pathology International

263

164

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Amyloid plaques and neurofibrillary tangles (NFTs) in the brain are the neuropathological hallmarks of Alzheimer's disease (AD). Amyloid plaques are composed of β-amyloid peptides (Aβ), while NFTs contain hyperphosphorylated tau proteins. Patients with familial AD who have mutations in the amyloid precursor protein (APP) gene have either increased production of Aβ or generate more aggregation-prone forms of Aβ. The findings of familial AD mutations in the APP gene suggest that Aβ plays a central role in the pathophysiology of AD. Aβ42, composed of 42 amino acid residues, aggregates readily and is considered to form amyloid plaque. However, the processes of plaque formation are still not well known. It is generally thought that Aβ is secreted into the extracellular space and aggregates to form amyloid plaques. Aβ as extracellular aggregates and amyloid plaques are thought to be toxic to the surrounding neurons. The intraneuronal accumulation of Aβ has more recently been demonstrated and is reported to be involved in synaptic dysfunction, cognitive impairment, and the formation of amyloid plaques in AD. We herein provide an overview of the process of the intraneuronal accumulation of Aβ and plaque formation, and discuss its implications for the pathology, early diagnosis, and therapy of AD.

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Section: The Amyloid Precursor Protein (App) and The Production Of Aβmentioning

confidence: 99%

Plaque formation and the intraneuronal accumulation of β‐amyloid in Alzheimer's disease

Takahashi

Nagao

Gouras

2017

Pathology International

263

164

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“…For imported orphan drugs, further promote the use of real-world research, an orphan drug import tariff reduction system and a free public platform for all stakeholders to share information on orphan drugs also need to be developed. International cooperation will play a crucial role in the response to rare diseases [ 33 ]. Besides, the high market accessibility rare diseases (endocrine, nutritional and metabolic diseases, etc.)…”

Section: Discussionmentioning

confidence: 99%

Evaluating the national system for rare diseases in China from the point of drug access: progress and challenges

Qiao

Liu

Shang

et al. 2022

Orphanet J Rare Dis

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Background There are about 7000 rare diseases worldwide, of which only 5% of the diseases can be treated with medicines, showing that it’s important to improve patient access to orphan drugs. Recently, China has actively worked to set up a national system for rare diseases to improve the diagnosis and treatment capabilities and ensure the accessibility of drugs. However, the benefits of the system have yet not to be measured. This study aimed to provide an overview of orphan drug access based on the Compendium of China’s First List of Rare Diseases and National Network to Collaborate on Diagnosis and Treatment of Rare Diseases, expecting to map a blueprint for orphan drug access in China. Methods Framework of China’s national system for rare diseases was summarized. We surveyed the availability and affordability of 79 approved orphan drugs based on the Compendium of China’s First List of Rare Diseases in 30 leading provincial institutions from 2017 to 2020. The availability was measured annually at 3 levels (market, hospital and drug), and affordability was reflected by comparing costs of daily defined dose with per capita income of urban and rural residents, with the National Basic Medical Insurance considered. Results The market availability of orphan drugs in China showed an upward trend. As of 2020, the median hospital-level availability was 41.1% (increased by 1.5 times), highly available drugs increased by 16.5%. There were 64/74 orphan drugs that were affordable to rural/urban residents with the National Basic Medical Insurance considered (an increase of 14.1%), and the urban–rural gap of affordability ratio was narrowed (down by 6.0%). Comprehensive analysis showed the proportions of drugs with better availability and affordability in urban and rural areas by 2020 were 39.4% and 32.3%, respectively, which had increased but were still at a low level. Conclusions China’s national system for rare diseases has made great progress in orphan drug access, indicating that it’s been functioning under the joint reformation of medical treatment, medical insurance and medicines supply. The list of rare diseases will be updated and collaboration in networks will be enhanced to further improve the system.

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“…rare diseases make these diseases a priority for policymakers, researchers, legislators, and healthcare professionals (4,5).…”

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confidence: 99%

Network established to collaborate on diagnosis and treatment of rare diseases in China: A strategic alliance backed by tiered healthcare is the key to the future

Ren

Wang

2019

IRDR

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China to further combat rare diseases (1). The network consists of 324 hospitals nationwide, including 1 leading national institution, 32 leading provincial institutions, and 291 member institutions in accordance with recommendations from provincial health authorities and a panel of experts. Worldwide, there are between 6,000 and 8,000 rare diseases. Eighty percent of rare diseases have identified genetic origins, 75% of rare diseases affect children, and 30% of patients with rare diseases die before the age of 5 (2,3). Rare diseases are serious chronic diseases and may be life-threatening. The features of rare diseases and the increasing number of identified

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The Government's role in regulating, coordinating, and standardizing the response to Alzheimer's disease: Anticipated international cooperation in the area of intractable and rare diseases

Cited by 6 publications

References 18 publications

Plaque formation and the intraneuronal accumulation of β‐amyloid in Alzheimer's disease

Plaque formation and the intraneuronal accumulation of β‐amyloid in Alzheimer's disease

Evaluating the national system for rare diseases in China from the point of drug access: progress and challenges

Network established to collaborate on diagnosis and treatment of rare diseases in China: A strategic alliance backed by tiered healthcare is the key to the future

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