Delving into the Latest Updates on Beyond Batten Disease Foundation with Synapse

概览

疾病领域	数量

内分泌与代谢疾病	1
神经系统疾病	1

排名前五的药物类型	数量

小分子化药	1

排名前五的靶点	数量

UGCG(葡萄糖神经酰胺合成酶)	1

关联

1

项与 Beyond Batten Disease Foundation 相关的药物

Miglustat/Trehalose

靶点

UGCG

作用机制

UGCG抑制剂

在研机构

Theranexus SA [+1]

原研机构-

在研适应症

神经元蜡样质脂褐质沉积症

非在研适应症-

最高研发阶段临床1/2期

首次获批国家/地区-

首次获批日期-

2

项与 Beyond Batten Disease Foundation 相关的临床试验

NCT05174039 / Active, not recruiting临床1/2期IIT

An Open-label Safety, Pharmacokinetic, and Efficacy Study of the Combination of Miglustat for the Treatment of CLN3 Disease in Patients 17 Years of Age and Older

This is an open label study in approximately 6 subjects in 2 centers to assess the safety, PK, and efficacy of the maximum tolerable dose (MTD) of oral miglustat (100 mg once daily [QD] to 200 mg 3 times daily [TID]) in subjects ≥ 17 years of age with CLN3 disease over a period of 104 weeks.

开始日期2022-02-02

申办/合作机构

Beyond Batten Disease Foundation

[+1]

NCT01793168 / RecruitingN/A

Coordination of Rare Diseases at Sanford

CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases. This program allows patients and researchers to connect as easily as possible to help advance treatments and cures for rare diseases. The CoRDS team works with patient advocacy groups, individuals and researchers to help in the advancement of research in over 7,000 rare diseases. The registry is free for patients to enroll and researchers to access. Visit sanfordresearch.org/CoRDS to enroll.

开始日期2010-07-01

申办/合作机构

Sanford Health Corp.

[+9]

100 项与 Beyond Batten Disease Foundation 相关的临床结果

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0 项与 Beyond Batten Disease Foundation 相关的专利（医药）

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1

项与 Beyond Batten Disease Foundation 相关的文献（医药）

2024-01-01·Palliative Care and Social Practice

Behaviours and psychological symptoms of childhood dementia: two cases of psychosocial interventions

Article

作者: Morris, Thomas ; Atee, Mustafa ; Lloyd, Rebecca ; Whiteman, Ineka

Childhood dementias are a group of rare, fatal neurodegenerative disorders, characterised by global cognitive decline, loss of previously acquired developmental skills and behaviours and psychological symptoms of dementia (BPSD). Batten disease, or neuronal ceroid lipofuscinosis, and Sanfilippo syndrome, or mucopolysaccharidosis type III, are two of the more common forms of childhood dementia disorders worldwide. While psychosocial interventions are the best available therapeutic approach for BPSD management in adult-onset dementia, there is very limited literature or clinical experience in the context of childhood dementia. To address this gap, we conducted a descriptive case analysis of BPSD profiles, associated contributing factors and targeted psychosocial interventions in two cases with childhood dementia disorders (Sanfilippo syndrome and CLN3 (juvenile onset) Batten disease) who were referred to Dementia Support Australia, a national dementia behaviour support service in Australia. Primary BPSD identified in these disorders included physical and verbal aggression and irritability/lability. In these cases, contributing factors to the development of BPSD were not monolithic, encompassing pain, caregiver’s approach and over or under-stimulation. Improvement in BPSD were observed using the Neuropsychiatric Inventory-Quesionnaire and globally noted as per the qualitative feedback reported by family and caregivers. Person-centred, multimodal psychosocial interventions were recognised as effective therapies in resolving BPSD in these cases. In conclusion, the case studies described the nature and presentation of BPSD in two common forms of childhood dementia and demonstrated the potential benefits of person-centred psychosocial interventions (delivered through national dementia-specific support programs) in alleviating BPSD such as irritability and aggression in these disorders.

100 项与 Beyond Batten Disease Foundation 相关的药物交易

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100 项与 Beyond Batten Disease Foundation 相关的转化医学

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