From 19 to 23 September 2017
At : Rome, Italy
The International Summer school intend i) to promote the establishment of Findable, Accessible, Interoperable, Reusable (FAIR) registries in compliance with IRDiRC and EU Recommendations and ii) to support cooperation among different registry stakeholders and coordination with registries that are developed within European Reference Networks and National Plans in the EU.
From 26 to 28 September 2017
At : Ljubljana, Slovenia
A 3-day course on NGS Ljubljana, Slovenia in the period of 25-27 October 2017. The focus of the course is on clinical diagnostics using exome/genome sequences, variant identification and analysis including afternoon practical sessions.
From 11 to 15 October 2017
At : Heidelberg, Germany
From 17 to 19 November 2017
At : Barcelona, Spain
Further information on the courses can be found at www.rrd-foundation.org
ASTERIX End Symposium
Date: 18-19 September 2017
Venue: Zaandam, Netherlands
For further information
2017 Rare Neuro-Immune Disorders Symposium
Date: 20 September, 2017
Venue: Ohio, United States
An education and advocacy conference for families, caregivers and individuals diagnosed with Acute Disseminated Encephalomyelitis (ADEM), Neuromyelitis Optica Spectrum Disorder (NMOSD), Optic Neuritis (ON), and Transverse Myelitis (including the subtype Acute Flaccid Myelitis). For further information
1st European Conference on Innovation in Paediatrics “Shaping the future of paediatrics”
Date: 20 - 22 September, 2017
Venue: Rome, Italy
This conference will be at the intersection of paediatrics with different disciplines and technology. It is dedicated to paediatricians, researchers, nurses, patients, entrepreneurs and specialists in in the field of innovation in paediatrics, especially in Europe. For further information
Shaping the Future of Pediatrics
Date: 20 – 22, September 2017
Place: Rome, Italy
It is dedicated to pediatricians, researchers, nurses, patients, entrepreneurs and specialists in the field of innovation in pediatrics, especially in Europe. For further information
Epidermolysis Bullosa 2017
Date: 24-27 September, 2017
Venue: Salzbourg, Austria
For further information
International Ataxia Research Conference IARC
Date: 27-30 September, 2017
Venue: Pisa, Italy
GoFAR, FARA and Ataxia UK in collaboration host what will be the second edition IARC where the latest research on 'Friedreich's ataxia will be presented and discussed. SCA and other progressive ataxias will also be presented and discussed. Plenary sessions will include the progress in the field of gene therapy, animal and cellular models, approaches pharmacologists and their development and clinical research. For further information
DISORDER – The Rare Disease Film Festival
Date: 3 October, 2017
Venue: Boston, USA
For further information
22nd International Congress of the World Muscle Society
Date: 3-7 October, 2017
Venue: Saint-Malo, France
For further information
22q11 EUROPE 2nd ALLIANCE MEETING
Date: 7-8 October, 2017
Venue: Dublin, Ireland
The theme of the meeting is Transition and our very own YEEP (Young Experts by Experience Panel) group will open the meeting who will speak about their own experiences of transition. For further information
49th Congress of the International Society of Paediatric Oncology
Date: 12-15 October, 2017
Venue: Washington, United States
For further information
Intracranial Hypotension Symposium
Date: 14 October, 2017
Venue: Santa Monica, United States
For further information
67th Annual Meeting of the American Society of Human Genetics
Date: 17-21 October, 2017
Venue: Florida, United States
The ASHG Annual Meeting is the largest human genetics meeting and exposition in the world. This year’s meeting is expected to attract over 6,500 scientific attendees, plus almost 250 exhibiting companies. The meeting provides a forum for the presentation and discussion of cutting-edge science in all areas of human genetics. For further information
2017 Rare Neuro-Immune Disorders Symposium
Date: 20-21 October, 2017
Venue: Columbus, United States
For further information
Towards Transformative Therapies for Sickle Cell Disease
Date: 24 October, 2017
Venue: New York, United States
For further information
5th Annual International Erdheim-Chester Disease Medical Symposium
Date: 25-27 October, 2017
Venue: New York, United States
The ECD Medical Symposium is the only medical conference focused on uniting medical professionals from around the world to share scientific findings, patient outcomes and case studies, and new developments in Erdheim-Chester disease. The annual meeting facilitates collaboration among physicians specializing in different therapies interested in learning more about ECD. Abstracts for presentations are due August 1, 2017. Email Jessica Corkran at jessica.corkran@erdheim-chester.org for more information. For further information
5th Annual International Erdheim-Chester Disease Patient & Family Gathering
Date: 27 October, 2017
Venue: New York, United States
The ECD Patient & Family gathering is the only conference focused on uniting those affected by Erdheim-Chester Disease from around the world to increase their knowledge of ECD, learn how to advocate for themselves in a healthcare system often unaware of their illness, and promote fellowship among patients and caregivers. Please email questions to Jessica Corkran at Jessica.corkran@erdheim-chester.org. For further information
3rd Annual Congress on Rare Diseases and Orphan Drugs
Date: 30 october - 1 November, 2017
Venue: San Antonio, United States
For further information
5th Glycoproteinoses International Conference
Date: 1-4 November, 2017
Venue: Roma, Italia
For further information
1st European ATTR Amyloidosis Meeting for Patients and Doctors
Date: 2-3 November, 2017
Venue: Paris, France
For further information
International Primary Immunodeficiences Congress 2017
Date: 8-10 November, 2017
Venue: Dubaï, United Arab Emirats
For further information
Orphan Drug Congress Europe
Date: 13-15 November, 2017
Venue: Barcelona, Spain
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
5th international conference focussing on translational medicine in inherited neuromuscular diseases
Date: 6 December, 2017
Venue: Ninds, United States 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. For further information
The ANR joins the Network of Centres of Excellence in Neurodegeneration (CoEN) to launch a Transnational Call aimed at supporting transnational collaborations in the field of neurodegenerative disease. Seven countries from the European Research Area and abroad are joining this call: Canada, France, Germany, Ireland, Italy, Spain and United Kingdom.
COEN initiative seeks to build productive links between research groups within recognised centres of excellence (CoEs) in neurodegeneration. The objective of this call is to stimulate new and unconventional approaches and creative solutions to the challenges of neurodegeneration research by undertaking high-risk / high-payoff research.
CoEN scope is broad. Projects may include studies to illuminate the understanding of neurodegenerative mechanisms, or create technological advances to support novel diagnostic or therapeutic approaches. Connectivity with research in related areas, such as inflammation and sensory neuroscience, or vascular and mental health is encouraged, as is outreach to experts in the fields of physical and computational science and industry when properly justified by the proposal.
DEADLINE: September 18, 2017
The French Foundation for rare diseases launches a call for proposals dedicated to ‘High throughput screening of therapeutic molecules’ to support projects aimed at identifying molecules with potential benefit in the treatment of rare diseases. Projects will be based on a high throughput/content screening (HTS/HCS) approach using compound libraries, towards the discovery of active molecules ‘hits’ with therapeutic potential. A second step towards investing into the ‘hit to lead’ process to approach drug-like characteristics will be supported.
DEADLINE: September 19, 2017
The Academy of Finland grants funding in open competition for research of the highest possible quality. We offer many different funding opportunities, and you can apply for funding to
The Academy Project funding scheme is designed to promote the quality and the diversity of research, scientific impact and impact beyond academia as well as science self-renewal. The aim is to attain internationally as high a scientific standard of work as possible and to support scientific breakthroughs and top-tier international
research collaboration.
DEADLINE: September 27, 2017
This funding opportunity announcement (FOA) seeks to expand knowledge and increase the evidence base for palliative care (PC) in advanced rare diseases, including rare cancers, and to improve physical and psychosocial well-being and quality of life among seriously ill individuals and their family caregivers.
DEADLINE: October 16, 2017
This Funding Opportunity Announcement (FOA) is to encourage Research Project Grant (R01) applications to pursue clinical observational (CO) studies to obtain data necessary for designing clinical trials for musculoskeletal, rheumatic, or skin diseases or conditions. Research data from observational cohort studies can enhance clinical trial design by providing essential information about disease symptoms, stages and timing of disease progression, comorbid conditions, availability of potential clinical trial participants, and outcomes that are important to patients. CO studies also can facilitate efforts to develop and/or validate objective biomarkers or subjective outcome measures for use in a future trial or trials. Applicants to this FOA are encouraged to propose studies that address significant obstacles or questions in the design of a clinical trial, such as determining the appropriate primary or secondary outcome measures, or identifying the stages of disease during which patients are most likely to respond to an intervention. Only observational studies will be supported by this FOA.
DEADLINE: November 1, 2017
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
In the field of rare diseases, understanding the physiopathological mechanisms and moreover, the evaluation of innovative therapeutic strategies, rely for a large part on animal models, particularly rodents. The translational research represents a link between exploratory steps of proofs of principle to clinical application in human. In some cases, the use of larger animal models is necessary to confirm the relevance of a concept or to demonstrate the feasibility, the efficiency and the safety of a therapeutic approach in conditions that mimic as closely as possible the human situation. The call for proposals launched by the French Foundation for rare diseases aims to directly support intermediary key steps towards clinical development for patients.
To complete and submit an application form, please access to the portal “Applicant portal”.
DEADLINE for pre-proposals: October 24, 2017 (5:00 pm)