Delving into the Latest Updates on Recordati Rare Diseases SARL 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

CPS1(氨基甲酰磷酸合成酶1)	1

关联

1

项与 Recordati Rare Diseases SARL 相关的药物

Carglumic acid

卡谷氨酸

靶点

CPS1

作用机制

CPS1激活剂

在研机构

Eurocept International B.V. [+6]

原研机构

Pola Chemical Industries, Inc.

在研适应症

异戊酸血症 [+4]

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

欧盟 [+3]

首次获批日期2003-01-24

100 项与 Recordati Rare Diseases SARL 相关的临床结果

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0 项与 Recordati Rare Diseases SARL 相关的专利（医药）

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2

项与 Recordati Rare Diseases SARL 相关的文献（医药）

2024-02-01·Future Cardiology

Real-world safety and efficacy of lomitapide in homozygous familial hypercholesterolemia: interim report of special-use survey in Japan

作者: Harada-Shiba, Mariko ; Kogawa, Noriaki ; Haruna, Shigenori

Drugs in Context

Mycosis fungoides-type cutaneous T-cell lymphoma (MF-CTCL) epidemiology and treatment pathway in Spain: new insights for an accurate description

Article

作者: D'Agostino, Paolo ; Sharp, Eric ; Kent, Alan ; Schmidt, Fabian ; Turini, Marco

BACKGROUNDMycosis fungoides-type cutaneous T-cell lymphoma (MF-CTCL) is a rare lymphoma localized in the skin. Due to its indolent nature and similarity to other skin conditions, diagnosis is often delayed or incorrect. Consequently, accurate calculations of incidence and prevalence are difficult to make. The treatment pathway taken by MF-CTCL patients can differ depending upon local healthcare systems, clinical policies and guidelines.AIMSThis study aims to (1) provide an estimate for the prevalence of treated MF-CTCL patients in Spain, (2) describe the Spanish patient treatment pathways for MF-CTCL, including quantification of the distribution of patients between primary, secondary and tertiary care institutions, and (3) investigate and quantify the treatment preferences of physicians.METHODOLOGYThis study employed primary market research methodologies to facilitate the collection of patient numbers and treatment practices from healthcare professionals (HCPs) and patients.LIMITATIONSPoor diagnosis of MF-CTCL may mean that actual prevalence levels in the broader population are higher than those estimated by this analysis of treated patients. This study was reliant upon accurate reporting by HCPs of patient numbers and their personal treatment practices. The rarity of the condition means the patient sample size is relatively small and limits possible accuracy of the quantitative analyses of patient-derived data, although this is supplemented by HCP-derived data in the analysis.FINDINGSAround 75% of MF-CTCL patients in Spain report that the initial diagnosis by their general practitioner is incorrect. This is usually due to underestimation of severity or type of skin disease. Once they have been correctly diagnosed (usually by a dermatologist) in secondary care, the management of MF-CTCL is led by dermatologists. In 39% of patients, shared care teams are also involved in patient management. Following diagnosis, the majority of patient management is conducted by secondary or tertiary care centers.CONCLUSIONSIncidence rates have increased in recent years, and possible reasons for this include improving levels of diagnosis. Survival in MF-CTCL has also increased over the last few decades. This trend appears to be reflected in the prevalence reported in this study, which is higher than suggested by some other estimates. However, it is still likely that there are further undiagnosed MF-CTCL patients in Spain due to the challenges of diagnosis at the primary care level.

100 项与 Recordati Rare Diseases SARL 相关的药物交易

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100 项与 Recordati Rare Diseases SARL 相关的转化医学

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