Wiskott Aldrich syndrome
- Eczema thrombocytopenia immunodeficiency syndrome
- Immunodeficiency 2
- IMD 2
- Aldrich syndrome
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ORDR Co-Sponsored Conferences
2016 Rare Disease Day at NIH, Monday, February 29, 2016
Location: Building 10, Masur Auditorium, National Institutes of Health, Bethesda, MD
Description: The 2016 Rare Disease Day at NIH will take place on February 29. The event, sponsored by NCATS and the NIH Clinical Center, aims to raise awareness about rare diseases, the patients they affect and the research collaborations that are addressing rare disease challenges. The day will feature tours, posters and exhibits, and presentations.
Primary Immune Deficiency Treatment Consortium Annual Scientific Meeting, Thursday, April 09, 2015
Location: Houston, TX
Description: The Specific Aim of this Workshop is to create a scientific forum for junior and senior investigators with a special interest in PID to focus on timely topics with respect to the immunobiology, diagnosis and therapeutic correction of PIDs leading to hypothesis-based prospective clinical trials that will optimize the care and durable correction of disease in children with PID.
Primary Immune Deficiency Treatment Consortium (PIDTC) Scientific Workshop, Thursday, May 01, 2014 - Saturday, May 03, 2014
Location: Seattle, WA
Description: The first three PIDTC Annual Scientific Workshops beginning in 2011 focused on critical topics for patients with SCID, Wiskott-Aldrich Syndrome (WAS) and Chronic Granulomatous Disease (CGD). Over 2012 and 2013, the Workshop has seen participation increase by more than 25% with a significant portion consisting of trainees and junior faculty interested in PID. We have also seen increasing participation from European colleagues resulting in the recent commitment to collaborate on PIDTC’s natural history study in CGD. Further discussion with European colleagues is underway to collaborate on PIDTC’s study of outcomes for patients with SCID who received HCT and have survived 25 years or more. Finally, the Workshops have facilitated the development of the first ever prospective multicenter intervention trial for newborns with SCID.
Gene Transfer and Rare Diseases Workshop, Thursday, September 13, 2012
Location: Rockville Hilton, Rockville, Maryland
Description: The NIH Office of Biotechnology Activities and Office of Rare Disease Research, NCATS, are co-sponsoring a workshop to discuss the advances in and challenges of gene transfer for rare diseases. There have been recent promising clinical successes in gene transfer protocols for rare diseases, such as retinal degeneration, hemophilia, and immune disorders. Investigators will discuss their results to date and opportunities and challenges for extending these successes to other similar rare diseases. The panel discussions will focus on strategies for maximizing opportunities for data sharing or platform development.
Primary Immune Deficiency Treatment Consortium (PIDTC) Scientific Workshop, Thursday, April 07, 2011 - Saturday, April 09, 2011
Location: San Francisco, CA
Description: This was a 3-day meeting. Participation at the workshop was by invitation and included representatives from each of the centers participating at PIDTC as well as representatives of CIBMTR, USIDNET, NIAID, ORDR, EBMT, and ESID. This was meant to favor collaboration and promote international collaborative trials in the field of these rare disorders. Special attention was paid to invitation of young investigators at a senior stage in their training or at the beginning of their academic careers. The meeting was open to representatives of the patient advocacy groups that are active in the field of primary immune deficiencies (PIDs) with the intent of promoting communication and collaboration. The results of the meeting will be published in a peer-reviewed journal.
The First Wiskott-Aldrich Syndrome Families and Investigators Conference, Friday, July 30, 2010 - Sunday, August 01, 2010
Location: Hilton Chicago Indian Lakes Resort and Conference Center, Bloomingdale, Illinois
Description: Conference goals were to (1) gather families affected by Wiskott-Aldrich syndrome (WAS) together in a nonmedical setting to share experiences with each other and present concerns to experts in immunology, BMT, and genetics; (2) review research achievements in WAS diagnosis, treatment (including BMT approaches and gene therapy), post-transplant outcomes (reconstitution and side effects of chemotherapy), and management of medical complications and family psychological issues; (3) develop and prioritize outstanding questions for future research; and (4) coordinate with planned efforts for a national WAS registry.
Primary Immunodeficiency Disease Consortium Conference, Thursday, June 11, 2009
Location: San Francisco, CA
Description: The CIS Primary Immune Deficiency Consortium Conference provided new investigators in this field the continued opportunity to enhance career development by learning from leaders in the field of primary immunodeficiency and other young investigators from institutions across the Americas and Europe.
Improving Cellular Therapy for Primary Immune Deficiency Diseases: Recognition, Diagnosis and Management, Thursday, May 21, 2009 - Friday, May 22, 2009
Location: EPN, 6130 Executive Blvd., Rockville, MD
Description: The first objective of this conference was to facilitate continued discussion and team building of pediatric hematopoietic cell transplant physicians and pediatric immunologists to work together to develop a North American consortium for treatment of children with PID. The workshop of May 2008 set in motion an important collaborative effort. This new workshop focused on challenging aspects of SCID/CID not previously addressed. Other objectives were to (2) conduct a survey of current practices in the workup and management of children with SCID/CID in preparation for HCT, (3) identify questions for future clinical research by utilizing the results of the survey, and (4) develop best current practice parameters (or guidelines) in this area. The document will be published in a respected peer-reviewed journal.