scholarly journals The Vienna Bone and Growth Center—care and research in the field of rare diseases

2021 ◽  
Vol 171 (5-6) ◽  
pp. 85-85
Author(s):  
Gabriele Haeusler ◽  
Rudolf Ganger ◽  
Roland Kocijan ◽  
Nadja Fratzl-Zelman
Keyword(s):  
Rare Diseases ◽  
Center Care ◽  
Growth Center

The Collaborative Network Approach: a model for advancing patient-centric research for Castleman disease and other rare diseases

2019 ◽  
Vol 3 (1) ◽  
pp. 97-105
Author(s):  
Mary Zuccato ◽  
Dustin Shilling ◽  
David C. Fajgenbaum
Keyword(s):  
Rare Diseases ◽  
Treatment Options ◽  
Castleman Disease ◽  
High Impact ◽  
Collaborative Network ◽  
Network Approach ◽  
Grant Applications ◽  
Research Questions ◽  
Novel Strategy ◽  
Patient Centric

Abstract There are ∼7000 rare diseases affecting 30 000 000 individuals in the U.S.A. 95% of these rare diseases do not have a single Food and Drug Administration-approved therapy. Relatively, limited progress has been made to develop new or repurpose existing therapies for these disorders, in part because traditional funding models are not as effective when applied to rare diseases. Due to the suboptimal research infrastructure and treatment options for Castleman disease, the Castleman Disease Collaborative Network (CDCN), founded in 2012, spearheaded a novel strategy for advancing biomedical research, the ‘Collaborative Network Approach’. At its heart, the Collaborative Network Approach leverages and integrates the entire community of stakeholders — patients, physicians and researchers — to identify and prioritize high-impact research questions. It then recruits the most qualified researchers to conduct these studies. In parallel, patients are empowered to fight back by supporting research through fundraising and providing their biospecimens and clinical data. This approach democratizes research, allowing the entire community to identify the most clinically relevant and pressing questions; any idea can be translated into a study rather than limiting research to the ideas proposed by researchers in grant applications. Preliminary results from the CDCN and other organizations that have followed its Collaborative Network Approach suggest that this model is generalizable across rare diseases.

Health care needs of patients with rare diseases

2004 ◽  
Author(s):  
R. M. A. van Nispen ◽  
P. M. Rijken ◽  
M. J. W. M. Heijmans
Keyword(s):  
Health Care ◽  
Rare Diseases ◽  
Health Care Needs ◽  
Care Needs

The Growth Center Model: Proactive programs of a university counseling service

1972 ◽  
Author(s):  
Melvin L. Foulds ◽  
James F. Guinan ◽  
David Hathaway ◽  
Robert Witchel ◽  
Fred Axelberd ◽  
...  
Keyword(s):  
Counseling Service ◽  
University Counseling ◽  
Growth Center

P 956. Next-Generation Sequencing in Patient Care—Arrived or Rejected in Rare Diseases?

2018 ◽  
Author(s):  
Angela Abicht ◽  
Teresa Neuhann ◽  
Stefanie Balg ◽  
Daniela Gonzalez-Fassreiner ◽  
Verena Steinke-Lange ◽  
...  
Keyword(s):  
Next Generation Sequencing ◽  
Patient Care ◽  
Rare Diseases ◽  
Next Generation ◽  
Generation Sequencing

Rare diseases and their patient organization: the Hungarian Federation of People with Rare and Congenital Diseases

2014 ◽  
Vol 155 (9) ◽  
pp. 329-333
Author(s):  
Gábor Pogány
Keyword(s):  
Social Services ◽  
Rare Diseases ◽  
Poor Quality ◽  
Congenital Diseases ◽  
Governmental Organization ◽  
Patient Organizations ◽  
Long Duration ◽  
Care And Support ◽  
Educational Professionals ◽  
Duration Of Hospitalization

The aim of the author is to discuss special issues of rare diseases, with emphasis on circumstances present in Hungary, including those leading to the foundation of the non-governmental organization, the Hungarian Federation of People with Rare and Congenital Diseases. The author briefly reviews the most important findings of current international surveys which have been performed with or without the involvement of member associations of the Hungarian Federation of People with Rare and Congenital Diseases. At the level of medical and social services in Hungary, it is still “incidental” to get to the appropriate expert or centre providing the diagnosis or treatment. It is difficult to find the still very few existing services due to the lack of suitable “pathways” and referrals. There are long delays in obtaining the first appointment, resulting in vulnerability and inequality along the regions. The overall consequence is the insufficiency or lack of access to medical and social services. There are also difficulties related to the supply of orphan medication and the long duration of hospitalization. At the level of patient organizations financial scarcity and uncertainty are typical, combined with inappropriate infrastructural background and human resources. The poor quality of organization of patient bodies along with insufficient cooperation among them are characteristic as well. The author concludes that a National Plan or Strategy is needed to improve the current fragmentation of services which would enable patients and health, social and educational professionals to provide and use the best care in the practice. This would ensure all patients with rare diseases to be diagnosed within a possible shortest time allowing access to the care and support needed in time resulting in a decrease in burden of families and society. Orv. Hetil., 2014, 155(9), 329–333.

Identifikasi Peran dan Strategi Pelabuhan Bebas Sabang

2020 ◽  
Vol 4 (3) ◽  
pp. 167-178
Author(s):  
Zurayna Sari
Keyword(s):  
Economic Development ◽  
Descriptive Analysis ◽  
Regional Growth ◽  
Maintenance Strategy ◽  
Regional Economic ◽  
Growth Center ◽  
Free Port ◽  
And Function ◽  
Role And Function

ABSTRAKPelabuhan berperan sebagai fasilitas penunjang pusat pertumbuhan regional dalam proses pembangunan ekonomi wilayah. Pelabuhan Bebas Sabang diarahkan sebagai pusat pertumbuhan ekonomi regional dan diharapkan dapat meningkatkan perekonomian Kawasan Sabang. Permasalahan yang dihadapi Pelabuhan Bebas Sabang adalah belum optimalnya peran dan fungsi Pelabuhan Bebas Sabang dalam menunjang perekonomian wilayah. Penelitian ini bertujuan untuk mengetahui peran Pelabuhan Bebas Sabang dalam mendorong perkembangan perekonomian Kawasan Sabang. Lingkup materi yang dibahas mencakup peran-peran Pelabuhan Bebas Sabang, menentukan potensi dan masalah serta upaya-upaya peningkatan peran Pelabuhan Bebas Sabang. Metode analisis yang dilakukan adalah analisis deskriptif dengan pendekatan analisis data kualitatif dan kuantitatif. Alat analisis yang digunakan adalah analisis SWOT IFAS-EFAS. Hasil analisis menunjukkan dalam kurun waktu 4 (empat) tahun terakhir dari tahun 2010-2013, Pelabuhan Bebas Sabang belum optimal dalam menjalankan perannya, sehingga membutuhkan strategi pengembangan dengan pendekatan Agressive Maintenance Strategy (strategi perbaikan agresif), yaitu strategi konsolidasi internal dengan memperbaiki faktor-faktor kelemahan untuk memaksimalkan pemanfaatan peluang.Kata kunci: Pengelolaan, SWOT IFAS-EFAS, WilayahABSTRACTPort was supporting facility of regional growth center in the process of regional economic development. Sabang free port was directed as the center of regional economic growth and expected to raise the economy of sabang. Problems faced by sabang free port was yet optimal role and function in supporting the economy of the region. This study aimed to determine the role of sabang free port in supporting the economic development of sabang. The covered material scope included roles of sabang free port, determining the potentials and problems and efforts of increasing the role of sabang free port. The method of analysis was descriptive analysis with qualitative and quantitative approach. The analytical tool used was the swot ifas-efas analysis. The analysis results showed in the period of 4 (four) years from 2010 until 2013, sabang free port was not optimal in carrying out its role yet, so it requires development strategies with agressive maintenance strategy approach, which is internal consolidation strategy by improving vulnerability factors to maximize the utilization of opportunities.Keywords:, Management, Regional, SWOT IFAS-EFAS

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