Courses & Educational Initiatives
Synaptic metabolism and brain circuitries in IEM: exploring old and new disorders
Date: 16 – 18 November, 2017
Venue: Barcelona, Spain
Further information on the courses can be found at www.rrd-foundation.org
Courses & Educational Initiatives and Events
Events
14th DIA Japan Annual Meeting
Date: 12-14 November, 2017
Venue: Tokyo Big Sight (Ariake), Japan
For further information
Orphan Drug Congress Europe
Date: 13-15 November, 2017
Venue: Barcelona, Spain
For further information
ELIXIR Innovation and SME Forum: Data Driven Innovation in Rare Diseases and Personalised Medicine
Date: 14-15 November, 2017
Venue: Paris, France
This ELIXIR Innovation event is focused on Rare Diseases and Personalised Medicine. Attendees will be immersed in a world of data-driven innovation, illustrated through talks by innovative companies and presentations of ELIXIR’s open data resources and services. With a mix of high-level keynote speakers and interactive sessions, this free event to will allow participants to discuss and interact with other companies and ELIXIR partners. To register: before the 8 November 2017
For further information
3rd Annual Boston Understand Your Genome® Conference
Date: 14 November, 2017
Venue: Massachusetts, United States
The Boston UYG is an interactive symposium in which approximately 75 leaders from the Boston business and academic communities will undergo whole-genome sequencing and explore their own genome as part of an all-day educational conference, and we invite you to join in on the exciting presentations, debates and comments from some of the most thought-provoking leaders in the field of sequencing, informatics and genomic medicine. For Further information
25th Fondation René Touraine Scientific Meeting 2017: Neuro-Immune Communication in the Skin
Date: 17 November, 2017
Venue: Paris, France
For further information
14th International Conference on Thalassaemia & Haemoglobinopathies and 16th TIF International Conference for Patients & Parents
Date: 17 - 19 November, 2017
Venue: Thessaloniki, Greece
For further information
Imaging in Neuromuscular Disease 2017
Date: 19-21 November, 2017
Venue: Berlin, Germany
For further information
DEBRA International Congress 2017
Date: 24 – 26 November, 2017
Venue: Wellington, New Zealand
The yearly DEBRA International Congress plays a crucial role in the push to one day see a cure for Epidermolysis Bullosa.For further information
5th international conference focussing on translational medicine in inherited neuromuscular diseases
Date: 27–29 November, 2017
Venue: Freiburg, Germany
The aim of this international conference is to share progress and lessons learned in the area of translational medicine in inherited neuromuscular diseases and plan for the delivery of future therapies to patients. For further information
European Conference on Rare Diseases 2018: Registrations and call for poster abstracts open
Date: 10-12 May, 2018
Venue: Vienna, Austria
The European Conference on Rare Diseases 2018 (https://www.rare-diseases.eu) Co-organised by EURORDIS (www.eurordis.org), DIA, and for the first time Orphanet, the theme of this edition of the conference will be "Rare Diseases 360°: Collaborative Strategies to leave no-one behind". The programme, registration and call for poster abstracts have now been published. Poster submissions are open until 31st January 2018. For further information
RE(ACT) congress
Date: 7-10 March, 2018
Venue: Bologna, Italy
The RE(ACT) Congress – The International Congress on Research of Rare and Orphan Diseases aims to bring together world leaders and young scientist from a variety of breaking through the scientific field to present cutting-edge research, to discuss results and to exchange ideas. Patients and patient organizations will also be present to share their experience during this 4th edition co-organized in collaboration with E-RARE ERA-NET. For further information
Grants
Call ‘NCL Research Award (50,000€)'
The NCL Foundation aims to help find a cure for the most prevalent form of childhood dementia caused by mutations in the CLN3 gene. This support an innovative pilot project at the Postdoctoral fellowship level. It is highly encouraging junior scientists, clinical researchers and medical fellows worldwide to submit projects that hold promise to help find and push forward therapies for CLN3 disease. They also highly encourage applicants that work in disease areas outside NCL, provided the proposed research is relevant to elucidate the role of CLN3 or find a cure for this disease. You can send your application via email to Research@ncl-foundation.com
Please, note that it is a 2-stage process. First of all, we need to receive your Letter of Intent before we accept your full application.
DEADLINE: November 30, 2017
Biological response modifier drugs for rare autoimmune diseases (NHS, UK)
The aim of the HTA Programme is to ensure that high-quality research information on the effectiveness, costs and broader impact of health technology is produced in the most efficient way for those who use, manage, provide care in or develop a policy for the NHS. Topics for research are identified and prioritised to meet the needs of the NHS. Health technology assessment forms a substantial portfolio of work within the National Institute for Health Research and each year about fifty new studies are commissioned to help answer questions of direct importance to the NHS. The studies include both primary research and evidence synthesis.
Research Question: What is the clinical effectiveness of biological response modifiers used for a range of rare autoimmune diseases and are they cost effective?
DEADLINE: November 30, 2017
NeuroNEXT Clinical Trials -U01 (NINDS, USA)
This FOA encourages applications for exploratory clinical trials of investigational agents (drugs, biologics, surgical therapies or devices) that may contribute to the justification for and provide the data required for designing a future trial, for biomarker validation studies, or for proof of mechanism clinical studies. Diseases chosen for study should be based on the NINDS’ strategic plan and clinical research interests (www.ninds.nih.gov/funding/areas/index.htm). Successful applicants will be given access to the NeuroNEXT infrastructure. Following peer review, NINDS will prioritize and order trials that are given access to the NeuroNEXT infrastructure. The NeuroNEXT Clinical Coordinating Center (CCC) will work with the successful applicant to efficiently implement the proposed study. The NeuroNEXT Data Coordinating Center (DCC) will provide statistical and data management support. The NeuroNEXT clinical sites will provide recruitment/retention support as well as on-site implementation of the clinical protocol.
Applicants do not need to be part of the existing NeuroNEXT infrastructure.
DEADLINES: December 6, 2017
Project Grant: Spring 2018 (CIHR, Canada)
The Project Grant program is designed to capture ideas with the greatest potential to advance health-related fundamental or applied knowledge, health research, healthcare, health systems, and/or health outcomes. It supports projects with a specific purpose and a defined endpoint. The best ideas may stem from new, incremental, innovative, and/or high-risk lines of inquiry or knowledge translation approaches.
The Project Grant program will:
– support a diverse portfolio of health-related research and knowledge translation projects at any stage, from discovery to application, including commercialization;
– promote relevant collaborations across disciplines, professions, and sectors; and,
– contribute to the creation and use of health-related knowledge.
DEADLINES for the letter of intent: February 6, 2018 and March 6, 2018 for application
Clinical studies of safety and effectiveness of orphan products research project grant (FDA, USA)
The goal of FDA’s OOPD grant program is to support the clinical development of products for use in rare diseases or conditions where no current therapy exists or where the product being developed will be superior to the existing therapy. FDA provides grants for clinical studies on safety and/or effectiveness that will either result in or substantially contribute to, market approval of these products. Applicants must include in the application’s Background and Significance section documentation to support the assertion that the orphan disease or condition to be studied is a “rare disease or condition” and an explanation of how the proposed study will either help support product approval or provide essential data needed for product development.
The grants are available to any foreign or domestic, public or private, for-profit or nonprofit entity (including State and local units of government). Federal agencies that are not part of the Department of Health and Human Services (HHS) may apply. Agencies that are part of HHS may not apply. For-profit entities must commit to excluding fees or profit in their request for support to receive grant awards. Organizations that engage in lobbying activities are not eligible to receive grant awards.
DEADLINE: February 7, 2018
Call ‘Neurodegeneration Award 2018'
The NCL Foundation is pleased to offer a new advancement training program. Thanks to great support from the Joachim Herz Stiftung they are able to announce another grant (100.000€). The so-called Neurodegeneration Award 2018 covers a postdoctoral fellowship salary for two years. The aim is to foster and improve synergies in research on the CLN3 childhood dementia and age-related neurodegeneration. Therefore, applicants must meet the following requirements:
- Two collaborating partners must co-apply for the fellowship
- One partner must actively engage in CLN3 research
- The second partner must be engaged in research on age-related neurodegeneration
Ideally, postdoctoral candidates should conduct a considerable part of their work in both laboratories. Laboratories interested in applying should first send a brief Letter of Intent. After acceptance, you will be asked to send in a full proposal. Whether you need help to find a collaborating partner do not hesitate to contact us.
You can send your application via email to Research@ncl-foundation.com
DEADLINE: February 28, 2018
Natural History Studies for Rare Disease Product Development: Orphan Products Research Project Grant (R01) (FDA, USA)
The objective of FDA’s Orphan Products Natural History Grants Program is to support studies that advance rare disease medical product development through characterization of the natural history of rare diseases/conditions, identification of genotypic and phenotypic subpopulations, and development and/or validation of clinical outcome measures, biomarkers and/or companion diagnostics. Applicants must include in the application’s Background and Significance section documentation to support that the estimated prevalence of the orphan disease or condition in the United States (US) is less than 200,000 (or in the case of a vaccine or diagnostic, information to support that the product will be administered to fewer than 200,000 people in the US per year), and an explanation of how the proposed study will either help support product approval or provide essential data needed for product development.
DEADLINES: August 31, 2018 for the letter of intent and October 15, 2018 for the application
EASL DANIEL ALAGILLE AWARD
EASL launched a brand new fellowship programme to encourage biomedical research in the field of paediatric and adult genetic cholestatic diseases for Young Investigators entitled 'The Daniel Alagille Award' in honour of Pr D.
Scientific award of 25,000 EUROS to support a research-based project. The EASL Daniel Alagille Award is generously sponsored by CTRS laboratories.
DEADLINE: 30 November, 2017
NeuroNEXT Clinical Trials
This FOA encourages applications for exploratory clinical trials of investigational agents (drugs, biologics, surgical therapies or devices) that may contribute to the justification for and provide the data required for designing a future trial, for biomarker validation studies, or for proof of mechanism clinical studies. Diseases chosen for study should be based on the NINDS’ strategic plan and clinical research interests (www.ninds.nih.gov/funding/areas/index.htm). Successful applicants will be given access to the NeuroNEXT infrastructure. Following peer review, NINDS will prioritize and order trials that are given access to the NeuroNEXT infrastructure. The NeuroNEXT Clinical Coordinating Center (CCC) will work with the successful applicant to efficiently implement the proposed study. The NeuroNEXT Data Coordinating Center (DCC) will provide statistical and data management support. The NeuroNEXT clinical sites will provide recruitment/retention support as well as on-site implementation of the clinical protocol.
DEADLINE: 6 December, 2017