Care for Rare Foundation

from Wikipedia, the free encyclopedia

The Care-for-Rare Foundation - Foundation for children with rare diseases at the Dr. von Haunersche Children's Hospital of the Ludwig Maximilians University in Munich was founded by the founders, the pediatrician Christoph Klein and the lawyer Andreas Staudacher, as a non-profit foundation under civil law under the patronage of Federal Minister Annette Schavan . On August 12, 2009, the legal recognition according to Section 80 of the German Civil Code (BGB) in conjunction with Section 5 of the Foundation Act from the Tübingen regional council as the competent foundation authority. In addition, the foundation is recognized as tax-privileged in the sense of fulfilling its charitable and benevolent purposes. In 2010 the foundation was the winner of the nationwide innovation competition 365 Landmarks in the Land of Ideas .

Motivation for founding

Children with rare diseases are among the weakest in society. Many rare diseases are incurable. It often takes several years before the correct diagnosis can be made. In view of the limited market capitalization of new products, the pharmaceutical industry has little interest in investing in the development of new drugs. The fundamentals of rare diseases in children can only be clarified through intensified research efforts. This knowledge can open up new therapeutic opportunities.

aims

  • Increasing the sensitivity to rare diseases
  • Financial support for poor families with children suffering from rare diseases - especially for patients from emerging and developing countries
  • Research into the causes of rare diseases
  • Development of innovative therapy strategies
  • Intensify a global dialogue to promote science and research in the field of rare diseases in children
  • Education and training of students, doctors and scientists

In contrast to many other humanitarian initiatives, the Care-for-Rare Foundation does not primarily support medical aid in individual cases, but pursues the goal of researching the fundamentals of the diseases and thus achieving a deeper understanding of the causes of the disease.

strategy

The foundation promotes internationally oriented interdisciplinary networks of doctors and scientists. It acts on a global level and particularly supports cooperation with developing and emerging countries. It cooperates with the Alliance of Chronic Rare Diseases (ACHSE eV), patient associations and various institutions and corporations in order to achieve its goals. The care-for-rare idea unfolds multidimensionally through the five funding lines.

Funding lines

Care-for-Rare Alliance

The Care-for-Rare Alliance is a large network of doctors and scientists who are committed to the foundation's vision - to cure all children worldwide with rare diseases. They work in children's clinics or at scientific institutes and thus support the foundation's mission: to treat children with rare diseases, to decipher the causes of the diseases and to develop therapies. The Care-for-Rare Center at Dr. von Haunersche Children's Hospital in Munich provides medical care for children with rare diseases. Centers of excellence work together around the world to support and heal children with rare diseases. Care-for-Rare Centers are currently being built at the following locations: at the University of Alexandria (Egypt), at the Sheiba Medical Center in Tel Aviv (Israel), at the National Center of Hematology in Karachi (Pakistan) and at the Wadia Childrens Hospital in Mumbai (India).

Care-for-Rare Academy

The Care-for-Rare Academy program supports colleagues around the world in learning about diagnoses and therapies for rare diseases. Scholarships are awarded in order to participate in selected congresses or to guarantee extended stays in competence centers. The call for tenders for the award of 10 scholarships is currently running at this year's international symposium for translational medicine "From Rare to Care" from 8. – 10. October in Herrenchiemsee.

Care-for-Rare Awards

The foundation offers two prizes every year to encourage young scientists to research in the field of rare diseases. The "Dr. Holger Müller" award, endowed with 5,000 euros, honors individual scientists or a group who have published an outstanding scientific article on the subject of "rare diseases" in the previous year. With up to 50,000 euros, the "Care-for-Rare Science Award" donated by the Werner Reichenberger Foundation provides great support for young scientists so that they can begin research projects in the field of rare diseases.

Care-for-Rare Aid

The Care-for-Rare aid enables donors worldwide to support individual patients in order to give them access to modern genomic medicine.

Care-for-Rare Awareness

The Care-for-Rare Awareness promotes the dissemination of information on rare diseases and leads awareness campaigns for the special needs of people with rare diseases.

Awards and award ceremonies

  • 2010 Prize winner in the nationwide innovation competition "365 Landmarks in the Land of Ideas"
  • awarded as "Science Foundation of the Year 2016" by the German University Foundation of the German University Association and the Dr. Jürgen Rembold Foundation
  • The foundation annually awards the Dr. Holger Müller Research Prize in cooperation with the Dr. Holger Müller Foundation . The aim of this award is to set an example for research into rare diseases . The namesake of the science award, Holger Müller (* September 14, 1948 - October 28, 2008), was a doctor of laboratory medicine and biochemistry and worked for 20 years as chief physician at the Klinik am Eichert in Göppingen. As a researcher he developed a. a. a method for the flow cytometric detection of HLA-B27 on lymphocytes .
    • The winner of the 2011 call is Karl Peter Schlingmann. The award ceremony took place on February 28, 2012. The outstanding work by Schlingmann on the genetic and pathophysiological basis of idiopathic infantile hypercalcemia was recognized .
    • The 2012 award winner is Frank Kaiser. At the award ceremony on March 7, 2013, Kaiser's work on mutations in the RAD21 gene as a genetic cause of cohesinopathies was recognized.
    • The 2013 winners are Daniel Kotlarz and Natalia Zietara. At the award ceremony on March 12, 2014, her work on identifying human interleukin-21 receptor deficiency as a new primary immunodeficiency disease was recognized.
    • The 2014 award winners are Theresa Schumacher and Lukas Bunse from the German Cancer Research Center in Heidelberg. At the award ceremony on March 12, 2015, her fundamental research into the development of a vaccine against brain tumors was recognized .
    • The 2015 award winners are Nina Bögershausen (Institute for Human Genetics at Göttingen University Medical Center) and I-Chun Tsai (Duke University, Durham, USA) for their research on the rare Kabuki syndrome. The award ceremony took place on March 16, 2016 in Esslingen.
    • The 2016 award winner is Peter Kühnen (Institute for Experimental Pediatric Endocrinology at the Charité - Universitätsmedizin Berlin), who receives the award for his research into the causes of genetically determined obesity.
  • In cooperation with the Werner Reichenberger Foundation, the Care-for-Rare Foundation annually awards the Care-for-Rare Science Award , endowed with 50,000 euros , in order to promote new and creative projects. An independent scientific panel of judges selects the project proposals according to criteria of scientific excellence, interdisciplinarity and clinical relevance.
    • The 2013 award winner is Tina Wenz for her project Towards a targeted protein replacement therapy for mitochondrial protein synthesis defects .
    • The 2014 award winner is Petra Wendler with the project submission Investigation of the Zellweger Syndrome Spectrum disorders at molecular level .
    • The 2015 award winner is Michael Schmeisser for the project submission Translational analysis of epilepsy in Phelan-McDermid syndrome and other Shankopathies
    • The 2016 award winner is Heymut Omran for the project submission Validity of diagnostic testing in Primary Ciliary Dyskinesia
    • The 2017 award winner is Christian Michael Grimm with the project submission Molecular dissection of the neuronal ceroid lipofuscinosis causing CLN3 protein
    • 2018 award winners are Tobias Hirsch and Dierk Niessing with the project submissions Center for cell and gene therapy in epidermolysis bullosa and graspPURA
    • The 2019 award winner is Holm Schneider with the project submission Prenatal therapy of X-linked hypohidrotic ectodermal dysplasia with a recombinant Fc-receptor targeted ectodysplasin-A1 replacement protein

organization

Board

Board of Trustees

Scientific Advisory Board

Web links

Individual evidence

  1. ↑ http://www.land-der-ideen.de/de/365-orte/preistraeger/care-rare-foundation  ( page no longer available , search in web archives )  Info: The link was automatically marked as defective. Please check the link according to the instructions and then remove this notice.@1@ 2Template: Toter Link / www.land-der-ideen.de  
  2. ^ German University Association: Science Foundation Prize. (No longer available online.) In: www.hochschulverband.de. Archived from the original on April 4, 2016 ; Retrieved April 4, 2016 . Info: The archive link was inserted automatically and has not yet been checked. Please check the original and archive link according to the instructions and then remove this notice. @1@ 2Template: Webachiv / IABot / www.hochschulverband.de
  3. ↑ Nationwide invitation to tender for the 2011 Dr. Holger Müller Prize ( Memento from October 21, 2013 in the Internet Archive ) In: innovations-report.de
  4. ↑ Press release: First award of the Dr. Holger Müller Research Prize for rare diseases in the old town hall in Esslingen 2, 2012 (PDF file)
  5. ↑ Dr. Holger Müller Research Award. In: aerzteblatt.de . Retrieved February 3, 2015 .
  6. ↑ KP Schlingmann, M. Kaufmann, S. Weber, A. Irwin, C. Goos, U. John, J. Misselwitz, G. Klaus, E. Kuwertz-Bröking, H. Fehrenbach, AM Wingen, T. Güran, JG Hoenderop, RJ Bindels, DE Prosser, G. Jones, M. Konrad: Mutations in CYP24A1 and idiopathic infantile hypercalcemia. In: The New England Journal of Medicine . Volume 365, Number 5, August 2011, ISSN  1533-4406 , pp. 410-421, doi : 10.1056 / NEJMoa1103864 , PMID 21675912 .
  7. ^ MA Deardorff, JJ Wilde, M. Albrecht, E. Dickinson, S. Tennstedt, D. Braunholz, M. Mönnich, Y. Yan, W. Xu, MC Gil-Rodríguez, D. Clark, H. Hakonarson, S. Halbach, LD Michelis, A. Rampuria, E. Rossier, S. Spranger, L. Van Maldergem, SA Lynch, G. Gillessen-Kaesbach, HJ Lüdecke, RG Ramsay, MJ McKay, ID Krantz, H. Xu, JA Horsfield, FJ Kaiser: RAD21 mutations cause a human cohesinopathy. In: American Journal of Human Genetics . Volume 90, number 6, June 2012, ISSN  1537-6605 , pp. 1014-1027, doi : 10.1016 / j.ajhg.2012.04.019 , PMID 22633399 , PMC 3370273 (free full text).
  8. ↑ D. Kotlarz, N. Ziętara, G. Uzel, T. Weidemann, CJ Braun, J. Diestelhorst, PM Krawitz, PN Robinson, J. Hecht, J. Puchałka, EM Gertz, AA Schäffer, MG Lawrence, L. Kardava , D. Pfeifer, U. Baumann, ED Pfister, EP Hanson, A. Schambach, R. Jacobs, H. Kreipe, S. Moir, JD Milner, P. Schwille, S. Mundlos, C. Klein: Loss-of- function mutations in the IL-21 receptor gene cause a primary immunodeficiency syndrome. In: The Journal of experimental medicine. Volume 210, number 3, March 2013, ISSN  1540-9538 , pp. 433-443, doi : 10.1084 / jem.20111229 , PMID 23440042 , PMC 3600901 (free full text).
  9. ↑ Scientific Advisory Board | www.care-for-rare.org. Retrieved January 23, 2018 .