Testimonials for Care-for-Rare

From our patients, colleagues, and supporters

Raz Somech

“Care-for-Rare is a great initiative! Established by clinician-scientists and empowered by a growing philanthropic community across the world, Care-for-Rare makes a true difference to children with rare diseases. I am proud to be a part of this inspiring project and wholeheartedly believe that it sets the stage for a new era of personalized medicine curing children with life-threatening diseases!”

~ Raz Somech, MD PhD

Chair, Department of Pediatrics, Sheba Medical Center, University of Tel Aviv, Israel

“Care-for-Rare provides a unique framework for us to further our work identifying the genetic causes of VEOIBD. We hope this will push our understanding of disease and eventually lead to the development of new cures for these very sick patients. Care-for-Rare has done an amazing job bringing together clinicians and scientists from around the world to push this forward.”

~ Alexio Muise

Professor, Department of Paediatrics and Biochemistry, University of Toronto

alexio in front of a mountain range
Bruce Beutler

“The destiny of children with rare diseases depends critically on enthusiastic scientists and international collaboration. Care-for-Rare provides a valuable platform for both — therefore, I proudly share its vision and mission!”

~ Bruce Beutler

UT Southwestern, Dallas,

Nobel Prize in Physiology of Medicine

Dr Penninger

“Care-for-Rare has a great mission — to make children with rare diseases, currently therapeutic orphans, the pioneers of a new chapter of medicine. In view of unprecedented opportunities to read genes and their function at a single cell level and to edit the human genome, Care-for-Rare inspires clinicians and scientists around the world to unremittingly tackle the causes of severe diseases and to nurture hope for patients and families by advancing science.”

~ Josef Penninger

Director, Life Sciences Institute, University of British Columbia

Professor, Medical University Vienna

“Science unremittingly seeks knowledge. This knowledge helps to alleviate suffering and to cure rare diseases. Care-for-Rare provides a framework for this mission.”

~ Prof. Dr. Harald zur Hausen

Nobel Laureate in Medicine

Harald zur Hausen nobel laureate

“Children with rare diseases need our solidarity and support. Our society will greatly benefit from expanding the frontiers of knowledge on rare diseases. Understanding genetics and genomics of rare diseases will pave the way to tailored precision medicine, not only for children with rare diseases, but also for adult patients with much more common diseases. Care-for-Rare thus supports cutting-edge science and medicine of high relevance — to alleviate suffering for children with rare diseases and to shape the future of medicine.”

~ Judy H Cho MD

Icahn School of Medicine at Mount Sinai, NY

Member, National Academy of Sciences USA

“The  Helmsley Charitable Trust supports Care-for-Rare as part of a worldwide initiative to help children with early onset inflammatory bowel disease. Our funds help both children and their families who are in dire need, and may also unlock clues to the disease to help develop new treatments and a cure for everyone with IBD. Dr. Klein and his team are connected to medical centers across the world, bringing hope to many children and their parents.”

~ Sandor Frankel

Trustee, The Leona M. and Harry B. Helmsley Charitable Trust

Sandy Frankel

“Care-for-Rare creates links and relationships between institutions, sciences, countries, and people. Most importantly, however, Care-for-Rare steers science towards the cause of children with rare diseases and thus provides an important framework to understand and ultimately cure life-threatening diseases.”

~ Hans Clevers

Professor at Utrecht University, Past-president of Royal Netherlands Academy of Arts and Sciences

Member, National Academy of Sciences USA​​

“The tragic loss of our son Jeffrey has prompted us to change the destiny of children with primary immunodeficiency diseases around the world. Care-for-Rare is a wonderful partner for this mission and Christoph Klein as spiritus rector together with his outstanding team has made remarkable progress.”

~ Vicki and Fred Modell

Jeffrey Modell Foundation in New York

modell family photo

knowah with his mom“In the Philippines, our doctors could not help our son Knowah, who was born with a rare immune disorder. Eventually, genetic testing in Seattle helped to make a clear diagnosis. Then Knowah received a life-saving stem cell transplant in Germany. All of this would not have been possible without the generous support by Care-for-Rare! I wish many more children in the world could benefit from doctors and scientists working together across boundaries!”

~ Knowah’s mother

“Stem cells can do everything. If we can correct defective stem cells, we can cure diseases. Care-for-Rare works with national and global leaders to provide cutting-edge cures to children with rare diseases regardless of their origin — this philanthropic mission deserves our support!”

~ Hans-Peter Kiem, MD PhD

Stephanus Family Endowed Chair for Cell and Gene Therapy, Fred Hutchinson Cancer Center, Seattle, WA

President-Elect, American Society of Gene and Cell Therapy

“I have been caring for children with primary immunodeficiencies for over 30 years and will do my best to help them with all possible partners, including Care-for-Rare.”

~ Yu Lung Lau

Chair and Professor of Department of Paediatrics and Adolescent Medicine,

University of Hong Kong

Yu Lung Lau Hong Kong

“In Astrophysics, the scientific community has a long-standing history of successful international collaboration. I have no doubt that children with rare diseases would greatly benefit from a strong spirit of collaboration. Care-for-Rare provides such an innovative framework to connect doctors and scientists around the world. With their help, children from the US and many other countries will get access to transformative science and cutting-edge clinical care!”

~ Reinhard Genzel

Max Planck Society and University of California, Berkeley, CA

Nobel Prize in Physics

Reinhard Genzel 2018 (KH9A9951-CCs)