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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Cydan Development, Inc., an orphan drug accelerator dedicated to creating therapies that improve the lives of people living with rare genetic diseases, today announced its support for Rare Disease Day by making a gift to newly formed Children’s National Rare Disease Institute (CNRDI). This gift will go toward the CNRDI’s Innovation Fund, which supports new projects including diagnostics, devices, research, therapeutics, and process engineering, which will help advance the care and experience for children with rare diseases. “We are grateful for Cydan’s support of the Children’s National Rare Disease Institute,” said Marshall Summar, MD, chief of Genetics and Metabolism at Children’s National and chairman of the board at the National Organization for Rare Disorders (NORD). “With philanthropic investments from companies like Cydan, we will redefine the standard of care for children with genetic disorders, especially those largely unknown to the general medical community.” Chris Adams, Ph.D., co-founder and chief executive officer at Cydan, said, “We are proud to support Children’s National and this first-of-its-kind center in the U.S. focused exclusively on advancing the care and treatment of children and adults with rare genetic diseases. Rare diseases affect approximately 30 million Americans, and it’s heartening to see more resources dedicated to the identification, treatment and cure of these disorders.” As one of the first philanthropic contributions to the CNRDI Innovation Fund, Cydan’s gift will help to serve as a catalyst for rare disease care initiatives and help create an ever-expanding clinical knowledge base that advances the testing and development of rare disease therapies. Rare Disease Day® is an annual awareness day celebrated worldwide. This day is dedicated to elevating public understanding of rare diseases and calling attention to the special challenges faced by patients and their families. Rare Disease Day takes place every year on the last day of February to underscore the scientific, medical and social issues that affect this population. It was established in Europe in 2008 by EURORDIS, the organization representing rare disease patients in Europe, and is now observed in more than 80 nations. Rare Disease Day is sponsored by the National Organization for Rare Disorders® (NORD®), a leading independent, nonprofit organization committed to the identification, treatment, and cure of rare diseases. Cydan is an orphan drug accelerator dedicated to delivering therapies that will significantly improve the lives of people living with rare genetic diseases. Cydan evaluates experimental new therapies and advances those with the highest potential to be disease modifying treatments. Cydan’s goal is to launch new companies focused on developing promising therapies for rare genetic diseases with high unmet medical need. Vtesse, Cydan’s first new company, was launched in January 2015 and is developing a novel therapy for Niemann-Pick Disease Type C (NPC). Imara was launched in 2016 and is developing a novel treatment for Sickle Cell disease. Cydan was founded in 2013 by a management team with extensive drug discovery, clinical development and business development experience and financed by leading life sciences investors NEA, Pfizer Venture Investments, Lundbeckfond Ventures, Bay City Capital and Alexandria Venture Investments. The accelerator is based in Tech Square in Cambridge, Mass. For more information, visit http://www.cydanco.com or contact Cydan at info@cydanco.com. Children’s National Health System, based in Washington, DC, has been serving the nation’s children since 1870. Children’s National is ranked in the top 20 in every specialty evaluated by U.S. News & World Report; one of only four children’s hospitals in the nation to earn this distinction. Designated a Leapfrog Group Top Hospital and a two-time recipient of Magnet® status, this pediatric academic health system offers expert care through a convenient, community-based primary care network and specialty outpatient centers. Home to the Children’s Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children’s National is one of the nation’s top NIH-funded pediatric institutions. Children’s National is recognized for its expertise and innovation in pediatric care and as a strong voice for children through advocacy at the local, regional and national levels. For more information, visit ChildrensNational.org.


WASHINGTON, Nov. 29, 2016 /PRNewswire-USNewswire/ -- Children's National Health System announces the launch of a major three-year research study of undiagnosed diseases in partnership with the Regeneron Genetics Center (RGC, a subsidiary of Regeneron Pharmaceuticals, Inc.). The research...


Gergen P.J.,National Institute of Allergy and Infectious Diseases | Teach S.J.,Childrens National Health System | Togias A.,National Institute of Allergy and Infectious Diseases | Busse W.W.,University of Wisconsin - Madison
Journal of Allergy and Clinical Immunology: In Practice | Year: 2016

Asthma exacerbations are important components of asthma morbidity. The Inner-City Asthma Consortium was established in the early 1990s to identify risk factors for and to evaluate treatments to reduce asthma symptoms and exacerbations. Early studies identified atopy and inadequate treatment as important drivers of asthma morbidity. Later studies demonstrated that good adherence to guidelines-based asthma care could virtually eliminate symptoms and reduce but not eliminate exacerbations. Looking at exacerbations by season, risk factors were found to vary across the different seasons. Of the 7 factors identified, allergic status and pulmonary functions were found to be important for exacerbations in all seasons, but allergy had its strongest effect in the fall season. Therefore, additional therapy directed at reducing the role of allergy was evaluated and found to significantly reduce exacerbations even in participants with good symptom control when receiving guidelines-based therapy. Despite this year around aggressive therapy, exacerbations remain albeit at a lower level and with less seasonal variation. Another strategy, the short term use of therapy aimed at reducing the role of allergy begun before the fall season and focused on individuals at high risk for exacerbations, was found to be an effective approach to minimize exacerbations and to limit the amount of therapy necessary. © 2015 Elsevier Inc.


WASHINGTON--(BUSINESS WIRE)--Emerging data from Children’s National Health System are providing hope that a new approach to cell therapy may effectively harness the cancer-killing potential of the natural immune system as a treatment for patients with blood and bone marrow cancers for whom stem cell transplantation has not worked. These patients typically have few treatment options and experience very high mortality rates. Initial results from the Phase 1 RESOLVE trial, a multi-institutional, Phase 1 dose-ranging study co-led by Kirsten Williams, M.D., and Catherine Bollard, M.D., M.B.Ch.B., Chief of the Division of Allergy and Immunology and Director of the Program for Cell Enhancement and Technologies for Immunotherapy (CETI) at Children’s National, showed that the majority (78 percent) of patients responded to multi tumor-associated antigen specific lymphocytes (TAA-L) treatment, and 44 percent of patients achieved complete remission with limited toxicity. The trial included patients diagnosed with one of four tumors who were treated with TAA-L upon relapse of disease post stem cell transplant. “These initial findings suggest that non-genetically engineered antigen-specific lymphocytes can be isolated, expanded and adoptively transferred to severely ill patients with active disease and positively impact tumor regression,” says Dr. Bollard. “We are encouraged by the promise of these data, which support similar efforts to exploit the immunotherapeutic potential of the natural T-cell repertoire.” The data, presented at the BMT Tandem meeting in Orlando, Florida, was a collaboration between investigators at Children’s National and researchers at Johns Hopkins University and Baylor College of Medicine who are testing a novel approach to treating cancer. Their approach includes introducing TAA-L – a natural T-cell subtype from the immune system – to high-risk patients with advanced hematologic or blood cancers including acute myeloid leukemia (AML)/ Myelodysplastic Syndromes (MDS), B-cell acute lymphoblastic leukemia (ALL) and Hodgkin lymphoma. The trial evaluates the safety and efficacy of both donor and patient-derived TAA-L as a novel treatment for patients with AML/MDS, B-cell ALL or Hodgkin lymphoma who are in active disease relapse pre- or post-allogeneic hematopoietic stem cell transplantation (HSCT). Patients with refractory and relapsed AML, MDS, ALL and Hodgkin lymphoma often have extremely poor clinical outcomes. For patients with these malignancies who relapse after allogeneic stem cell transplantation, the prognosis is even more dismal. Sadly, one-year mortality rates for this population approach 90 percent. The results include patients receiving adoptively transferred TAA-L manufactured cells across all dosing cohorts allowed per protocol (dose levels one through four), 10 patients in total. Preliminary observations conclude that ex vivo manufactured TAA-L, composed primarily of central effector memory T-cells, can be successfully isolated and expanded to clinically relevant numbers, cryopreserved, and safely infused to patients after relapse of disease. There were no cases of graft-versus-host disease or other autoimmune-mediated toxicity reported, and there have been no observations of Cytokine Release Syndrome or neurotoxicity associated with TAA-L treatment. The RESOLVE trial has been and/or is funded by a National Institutes of Health grant (P01CA015396), the Leukemia Lymphoma Society, Ben’s Run and Hyundai Hope on Wheels. The BMT Tandem meeting is the combined annual meetings of the Center for International Blood & Marrow Transplant Research and the American Society for Blood and Marrow Transplantation. Children’s National Health System, based in Washington, DC, has been serving the nation’s children since 1870. Children’s National is ranked in the top 20 in every specialty evaluated by U.S. News & World Report; one of only four children’s hospitals in the nation to earn this distinction. Designated a Leapfrog Group Top Hospital and a two-time recipient of Magnet® status, this pediatric academic health system offers expert care through a convenient, community-based primary care network and specialty outpatient centers. Home to the Children’s Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children’s National is one of the nation’s top NIH-funded pediatric institutions. Children’s National is recognized for its expertise and innovation in pediatric care and as a strong voice for children through advocacy at the local, regional and national levels. For more information, visit ChildrensNational.org, or follow us on Facebook and Twitter.


Tosi L.L.,Childrens National Health System | Warman M.L.,Boston Childrens Hospital
Bone | Year: 2015

Rare bone diseases account for 5% of all birth defects and can cause significant morbidity throughout patients' lives. Significant progress is being made to elucidate the pathophysiological mechanisms underlying these diseases. This paper summarizes presentation highlights of a workshop on Rare Skeletal Diseases convened to explore how the study of rare diseases has influenced the field's understanding of bone anabolism and catabolism and directed the search for new therapies benefiting patients with rare conditions as well as patients with common skeletal disorders. © 2015.


News Article | March 1, 2017
Site: www.prnewswire.com

WASHINGTON, March 1, 2017 /PRNewswire-USNewswire/ -- First Lady Melania Trump today met with patients, families, doctors and other care team members at Children's National Health System, to talk about their personal experiences and health care journeys, and how the gift of nature and the...


Russell Cruz C.,Childrens National Health System | Bollard C.M.,Childrens National Health System
Haematologica | Year: 2015

Hematopoietic stem cell transplantation has revolutionized the treatment of hematologic malignancies, but infection, graft-versus-host disease and relapse are still important problems. Calcineurin inhibitors, T-cell depletion strategies, and immunomodulators have helped to prevent graft-versus-host disease, but have a negative impact on the graft-versus-leukemia effect. T cells and natural killer cells are both thought to be important in the graft-versus-leukemia effect, and both cell types are amenable to ex vivo manipulation and clinical manufacture, making them versatile immunotherapeutics. We provide an overview of these immunotherapeutic strategies following hematopoietic stem cell transplantation, with discussions centered on natural killer and T-cell biology. We discuss the contributions of each cell type to graft-versus-leukemia effects, as well as the current research directions in the field as related to adoptive cell therapy after hematopoietic stem cell transplantation. © 2015 Ferrata Storti Foundation.


Su L.,Childrens National Health System
Current Problems in Pediatric and Adolescent Health Care | Year: 2015

Since humans are an integral part of healthcare delivery, it is appropriate to understand how human nature and human error impact patient safety. A thorough understanding of the interactions between humans and the medical environment could help decrease errors that result in patient harm. This article describes some of our findings from a study that revealed unexpected behaviors which were documented on video during critical events (cardiac arrests) in a pediatric cardiac intensive care unit (CICU). We changed our training process for cardiac arrests as well as our nurse staffing in the CICU based upon results of our study. We believe that the lessons learned in our CICU are generalizable to both inpatient and ambulatory settings. We also emphasize the importance of collaborating with social scientists to rigorously study innate maladaptive patterns of human behavior to determine strategies to mitigate "human factors" during acute medical crises. © 2015 Mosby, Inc. All rights reserved.


Roberts R.J.,Childrens National Health System
Anesthesia and Analgesia | Year: 2016

Six Sigma and Lean methodologies are effective quality improvement tools in many health care settings. We applied the DMAIC methodology (define, measure, analyze, improve, control) to address deficiencies in our pediatric anesthesia supply chain. We defined supply chain problems by mapping existing processes and soliciting comments from those involved. We used daily distance walked by anesthesia technicians and number of callouts for missing supplies as measurements that we analyzed before and after implementing improvements (anesthesia cart redesign). We showed improvement in the metrics after those interventions were implemented, and those improvements were sustained and thus controlled 1 year after implementation. © 2016 International Anesthesia Research Society


Sady M.D.,Childrens National Health System | Vaughan C.G.,Childrens National Health System | Gioia G.A.,Childrens National Health System
Archives of Clinical Neuropsychology | Year: 2014

Psychometric characteristics of the Postconcussion Symptom Inventory (PCSI) were examined in both concussed (n = 633) and uninjured (n = 1,273) 5 to 18 year olds. Parent- and self-report forms were created with developmentally appropriate wording and content. Factor analyses identified physical, cognitive, emotional, and sleep factors; that did not load strongly or discriminate between groups were eliminated. Internal consistency was strong for the total scales (α = 0.8-0.9). Test-retest reliability for the self-report forms was moderate to strong (intraclass coeffecients, ICCs = 0.65-0.89). Parent and self-report concordance was moderate (r =. 44-.65), underscoring the importance of both perspectives. Convergent validity with another symptom measure was good (r =. 8). Classification analyses indicated greater discriminability from parent report, but caveats to this are presented. With strong psychometric characteristics, the four versions of the PCSI capture important postconcussion symptoms and can be utilized to track recovery from pediatric concussion and guide treatment recommendations. © The Author 2014. Published by Oxford University Press. All rights reserved.

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