The 14th Diamond Blackfan Anemia International Consensus Conference was a huge success. It is a privilege to continue the meeting founded and previously sponsored by Marie Arturi and the Daniella Maria Arturi Foundation. The DBA Foundation is grateful for the support of DBA Canada, the Daniella Maria Arturi Foundation, DBA-UK, Captain Courageous (Australia) and the Resource Capital Funds Foundation. The cooperation Continue Reading »Posted in: News, Research, Spotlight Stories
By: Steven R. Ellis, Ph.D. Dateline: August 21st 2015, Brussels Belgium Ding…………….that’s odd, it is 3:00AM back in the US: who would be e-mailing me now? Ah, it’s Jeff Lipton; who happens to be sitting immediately in front of me listening to a talk at the 10th triennial Conference on Ribosome Synthesis. The e-mail is sent to all members of Continue Reading »Posted in: News, Research, Spotlight Stories
The Diamond Blackfan Anemia Foundation (DBAF) and Diamond Blackfan Anemia Canada (DBAC) are joining forces once again to fund Johan Flygare, M.D., Ph.D.’s project entitled, “A novel group of kinase inhibitors for treatment of Diamond Blackfan Anemia.” The goal of this $35,000 project is to develop safer and more potent drugs to treat DBA. Ideally, these drugs would act by Continue Reading »Posted in: News, Research, Spotlight Stories
The Diamond Blackfan Anemia Foundation (DBAF) is pleased to support the hard work and research efforts of Steven R. Ellis, Ph.D. at the University of Louisville. Dr. Steve Ellis’ professional and personal commitment to DBA is immeasurable and deeply appreciated. Not only is Steve our dedicated (and unpaid) Research Director, who is working tirelessly behind the scenes, he also writes the DBAF’s e-newsletter Continue Reading »Posted in: News, Research, Spotlight Stories
The Diamond Blackfan Anemia Foundation (DBAF), in collaboration with DBA-UK, is pleased to announce funding for a project entitled, “Ribosome biogenesis, cellular signaling pathways and Diamond Blackfan anemia.” A $41,810 grant was awarded to Nicholas James Watkins, Ph.D, a senior lecturer in the Institute for Cellular and Molecular Biosciences at the University of Newcastle upon Tyne in the United Kingdom. Continue Reading »Posted in: News, Research, Spotlight Stories
The Diamond Blackfan Anemia Foundation (DBAF) is proud to support the Ribosome Synthesis Meeting held tri-annually. This international meeting, celebrating its 10th anniversary, brings together investigators from all over the world to focus on issues related to ribosome synthesis. The 2015 EMBO Conference on Ribosome Assembly will be held at the Hotel Metropole in downtown Brussels from August 19-23, 2015. The meeting will focus Continue Reading »Posted in: News, Research, Spotlight Stories, Upcoming Events
The Diamond Blackfan Anemia Foundation is proud to support the research of Dr. Shai Izraeli, Professor in the Department of Human Molecular Genetics and Biochemistry at Tel Aviv University and the Head of the Childhood Leukemia Research Institute within the Department of Pediatric Hematology/Oncology and the Cancer Research Center at Sheba Medical Center, Israel. The DBAF provided a $32,000 grant Continue Reading »Posted in: Misc, News, Research, Spotlight Stories
The Diamond Blackfan Anemia Foundation (DBAF), in partnership with DBA Canada (DBAC), is proud to provide a $25,000 grant to Dr. David Bodine for his project, Assay Development and Preliminary Screening of Small Molecules to Increase Ribosomal Protein Levels. David Bodine, PhD, Senior Investigator and Chief, Genetics and Molecular Biology Branch Head, Hematopoiesis Section of the National Human Genome Research Institute explained, “The vast Continue Reading »Posted in: News, Research
In April 2014 when the DBAF awarded Dr. Harvey Lodish $21,281, the intent was to fill a gap for a few months until Dr. Lodish received government money. Unfortunately, Dr. Lodish was notified that the Telemedicine & Advanced Technology Research Center program had been reorganized and discontinued due to lack of available funding from Congress, leaving him with no funds to Continue Reading »Posted in: News, Research, Spotlight Stories
The Diamond Blackfan Anemia Foundation is proud to support the very important work of Vijay G. Sankaran, M.D., Ph.D. of Boston Children’s Hospital. The DBAF has provided $36,000 to fund Dr. Sankaran’s research project entitled, Defining GATA1 Transcriptional Alterations in Diamond-Blackfan Anemia. In addition to his clinical responsibilities, Dr. Sankaran is an outstanding investigator and was a leader in the Continue Reading »Posted in: Misc, News, Research, Spotlight Stories
The Diamond Blackfan Anemia Foundation is pleased to announce the funding of another exciting research project. A grant for $35,000 was awarded to Daniel Finley, Ph.D. for his project entitled, “A new mode of ribosome regulation specific to reticulocytes.” Dr. Daniel Finley, a well-established investigator and a professor at Harvard University, is new to the DBA field. He will be Continue Reading »Posted in: Misc, News, Research
The Diamond Blackfan Anemia Foundation (DBAF) is proud to announce the funding of another important research project. Through the dedication, hard work, and generosity of our families, friends, and supporters, the DBAF was able to award $21,281 to Dr. Harvey Lodish’s project entitled, “High-throughput screening identifies many novel potential therapies for Diamond Blackfan Anemia.” The esteemed Dr. Lodish provided the Continue Reading »Posted in: News, Research, Spotlight Stories
The 13th Diamond Blackfan Anemia International Consensus Conference was held March 8th through the 10th at the Ritz-Carlton Hotel in downtown Atlanta, Georgia. This year’s conference saw a record number of abstracts accepted for presentation as either short talks or posters. This increased number of abstracts submitted relative to previous years, in part, reflects an increasing number of investigators entering Continue Reading »Posted in: ICC, News, Research, Spotlight Stories
The Diamond Blackfan Anemia Foundation is proud to partner with DBA Canada to fund a novel gene therapy research proposal from Dr. Johnson Liu, MD at The Feinstein Institute for Medical Research. The $48,210 project is titled, “Repairing Dominant Negative Mutation of RPS19 Gene by Spliceosome-Mediated RNA Trans-Splicing.” This approach takes advantage of the fact that human genes are made Continue Reading »Posted in: News, Research, Spotlight Stories
The DBA Foundation is excited to announce the funding of Dr. George Thomas’ research project entitled, “The role of p53 in mediating the anemic response in DBA patients with defects in RPL5 or RPL11.” Thank you to DBA Canada for their joint funding support to make this possible! The long-term goal of this study is to understand the mechanisms through Continue Reading »Posted in: News, Research, Spotlight Stories
We are so proud of Dr. Johan Flygare and bursting with excitement and gratitude! The DBAF has enthusiastically funded numerous of his earlier DBA research projects and this young researcher’s hard work and dedication have been rewarded! The Ragnar Söderberg Foundation recently announced the Ragnar Söderberg Fellow in Medicine 2013. Johan Flygare was one of the six scientists who received Continue Reading »Posted in: News, Research, Spotlight Stories
The Diamond Blackfan Anemia Foundation (DBAF) is pleased to support the hard work and research efforts of Steven R. Ellis, Ph.D. at the University of Louisville. Dr. Steve Ellis’ professional and personal commitment to DBA is immeasurable and deeply appreciated. Not only is Steve our dedicated (and unpaid) Research Director, who is working tirelessly behind the scenes, he also writes Continue Reading »Posted in: News, Research, Spotlight Stories
The Diamond Blackfan Anemia Foundation (DBAF) is pleased to announce the funding of another exciting research project. Dr. Jingping Ge, MD, PhD was awarded a $30,000 grant for her project entitled, “Role of TGF-beta signaling in the pathogenesis of DBA.” Dr. Ge’s preliminary data reveal that TGF-beta inhibits the reprogramming of differentiated cells to induced pluripotent stem cells (iPSCs) and even Continue Reading »Posted in: News, Research, Spotlight Stories
The Diamond Blackfan Anemia Foundation (DBAF) and Diamond Blackfan Anemia Canada (DBAC) have once again joined forces to fund a very exciting research project. Dr. John Crispino, PhD of Northwestern University was awarded a $50,000 grant for his project entitled, “Overriding GATA1 mutations in Diamond Blackfan Anemia.” The DBAF appreciates DBAC’s $15,000 contribution towards this project and is grateful to Continue Reading »Posted in: News, Research, Spotlight Stories
Dear DBA Patients and Families: We are pleased to announce the opening of: “The Use of Novel Therapies to Reconstitute Blood Cell Production and Promote Organ Performance, using Bone Marrow Failure as a Model: A Pilot, Phase II Study of the Amino Acid Leucine in the Treatment of Patients with Transfusion-Dependent Diamond Blackfan Anemia” Diamond Blackfan Anemia (DBA) is a Continue Reading »Posted in: Clinical Trials, News, Research, Spotlight Stories
Dr. Mitchell Weiss, Dr. Monica Bessler, Dr. Philip Mason and their entire team at The Children’s Hospital of Philadelphia have shown that skin cells from patients with DBA can be reprogrammed to produce blood cells. This breakthrough work is a step forward in understanding DBA and could lead to exciting new approaches in gene therapy and treatment options. For many years, Continue Reading »Posted in: News, Research
Dr. Harvey Lodish of the Whitehead Institute published new findings identifying a protein that is the target of prednisone or prednisolone, the drugs that are used to increase red blood cell production in some patients with Diamond Blackfan Anemia. The discovery could spur development of drugs capable of increasing this protein’s production without causing the severe side effects associated with Continue Reading »Posted in: News, Research
Congratulations to Stuart H. Orkin, MD, chairman of the department of pediatric oncology at Dana-Farber/Children’s Hospital Cancer Center. Dr. Orkin was recently awarded the Jessie Stevenson Kovalenko Medal from the National Academy of Sciences for his pioneering achievements in defining the molecular basis of blood disorders and the mechanisms governing the development of blood stem cells and individual blood lineages. Continue Reading »Posted in: News, Research
The DBAR has noted that many patients with DBA have issues that may be due to the hormone-producing gland (endocrine) system abnormalities, including short stature, thyroid dysfunction, delayed puberty in both boys and girls, and diabetes. We are interested in determining if these problems are due to long-term continuous steroid use, iron overload from transfusion therapy or from DBA itself. Continue Reading »Posted in: News, Research, Spotlight Stories
DBAF Funds More Research and a Research-rich Conference The DBA Foundation (DBAF) is proud to announce the funding of Dr. Fredrick Goldman’s research project entitled, Toward a cure for DBA: creation of patient-specific gene corrected hematopoietic stem cells using induced pluripotent stem cell technology. We are pleased to fund this exciting project that is on the cutting edge of research and has Continue Reading »Posted in: News, Research
The DBA Foundation (DBAF) is proud to announce the funding of Dr. Adrianna Vlachos’ research project entitled, A Stategic Approach to Gene Discovery in DBA. We are pleased to fund this exciting project with DBA Canada (DBAC) and appreciate their $25,000 commitment towards this $125,225 multi-centered project. Adrianna Vlachos, MD The goal of this research project is to identify genes affected in the Continue Reading »Posted in: News, Research
We are pleased to announce the funding of Dr. Johan Flygare’s project titled: Identification of Genetic and Chemical Modifiers of Erythropoiesis in Diamond Blackfan Anemia. This project is significant for many reasons. The Diamond Blackfan Anemia Foundation is grateful to Diamond Blackfan Anemia Canada (DBAC) for their generous support in helping to fund Dr. Flygare’s work. DBAC received charitable status only three months Continue Reading »Posted in: News, Research
Principal Investigator: Emanuela Tolosano, Co-Principal Investigator: Deborah Chiabrando This project is aimed at defining the role of the Feline Leukemia Virus, subgroup C, Receptor (FLVCR) in the pathogenesis of Diamond-Blackfan anemia (DBA). FLVCR encodes a protein that exports excess heme from cells. It has been suggested that defects in globin synthesis, perhaps as a result of ribosomal protein mutations, could result in Continue Reading »Posted in: News, Research
Dear DBA Patients and Families: We are pleased to announce the opening of the “Pilot Study of Lenalidomide (Revlimid) in Adult Patients with Red Blood Cell Transfusion-Dependent Anemia” at the Stanford University Medical Center. The rationale for evaluating lenalidomide in DBA comes from the study of an acquired form of bone marrow failure named myelodysplastic syndrome (MDS). Although DBA is Continue Reading »Posted in: News, Research
A Message from Ellen, DBA Nurse We need to find out how many are interested in the leucine trial so we can report back to the reviewers for funding. It is a slow process but we are about 3/4 of the way there. It already passed the pre- approval, proposal internal review, and proposal external review. Once it is approved, Continue Reading »Posted in: News, Research
DBAF is a proud co-sponsor of a Ribosome Synthesis Meeting held tri-annually. This meeting is International and brings together investigators from all over the world to focus on issues related to ribosome synthesis. The meeting alternates every third year between the US and Europe. The meeting in August 2009 will be held in Germany. In 2009, as was the case Continue Reading »Posted in: News, Research
Thanks to the support of our famiies and friends we were able to fund another worthwhile DBA research project. Dr. David M. Bodine from the NIH is being funded for his research to create and analyze mouse models for some of the mutations that cause Diamond-Blackfan Anemia (DBA). An animal model of DBA will allow an in depth analysis of Continue Reading »Posted in: News, Research
We would like to sincerely thank families and friends for your support and fundraising efforts. DBAF has recently been able to fund 3 research projects and a scientific conference. Because of your efforts more invaluable research is being supported to help find a better treatment and hopefully someday a cure for DBA. Principal Investigator: Dr. Johan Flygare Research: Characterizing the Continue Reading »Posted in: News, Research
We are not the first to say this and we will not be the last, the DBAF has saved our child’s life. I cringe at the thought of what her care would be had we not met the DBAF.
While I know the road will not always be without struggle, we will continue to learn more through research and the determination of the amazing DBA community.
This is David at 1 month old weighing almost 2 pounds. DBA changed our paths in life, but I will ALWAYS be there to hold his hand for he holds my heart.
We are hopeful that a cure will be discovered soon so that our son doesn’t have to endure pokes and disruptions and can feel good all the time – isn’t that what every child deserves?