Rare Diseases International 5th Annual Meeting
On 21 February 2019
At : New York, USA
Events
Organisation for Rare Diseases India's flagship event: Racefor7
On 25 February 2019
At : India and the USA
Rare Disease Day
On 01 March 2019
At : Worldwide
2nd International Congress on Advanced Treatments in Rare Diseases (RARE2019)
From 05 to 06 March 2019
At : Vienna, Austria.
9th Orphan Drugs & Rare Diseases Global Congress 2019 Europe
From 05 to 07 March 2019
At : London, United Kingdom.
Myology 2019
From 26 to 29 March 2019
At : Bordeaux, France
Duchenne Muscular Dystrophy Symposium
On 22 June 2019
At : Salzburg, Austria
International Primary Immunodeficiency Congress 2019
From 07 to 09 November 2019
At : Madrid, Spain
1st Global Congress on epidermolysis bullosa
From 20 to 24 January 2020
At : London, UK
Courses & Educational Initiatives
1rst workshop on ancient rare diseases
From 27 February to 01 March 2019
Venue : Berlin, Germany
EURORDIS Winter School 2019
From 12 to 16 March 2019
At : Imagine Institute, Paris, France
Applications are now open. Applicants will be notified of the outcome of their application by mid October.
Grants
DEBRA All priorities Research Call
DEBRA International is inviting research proposals for the ‘All-Priorities‘ Research Call. There is a one stage application process with a submission deadline by March 11, 2019.
The ‚All-Priorities‘ Research Project grants scheme calls for research proposals across DEBRA's four priority research areas:
- Improve our understanding of the biology and genetics of all forms of EB, as better understanding can lead to new approaches to diagnose and treat EB
- Work towards the development of therapies (including possible gene-therapies, cell-therapies, drug therapies or protein therapies)
- Understand the nature of wound healing and the development of skin cancer in EB, and seek to develop better treatments and prevention strategies
- Support clinical care research to improve the management of EB through symptom relief
Funding up to €270.000 over three years
Research proposals must be submitted online.
Closing date for applications Monday, March 11, 2018 at 17:00 GMT.
Histio UK call for proposals
Applications are invited for studies the histiocytic disorders including Histiocytic Lymphohistiocytosis (HLH), Langerhans Cell Histiocytosis, or rare histiocytic disorders for a maximum budget of £50,000 and duration of up to 24 months.
Histio UK will consider scientific and clinical research on the epidemiology, pathogenesis, diagnosis, treatment and clinical care of patients with histiocytosis.
Applicants will be required to describe the likely impact of their research upon patients with histiocytosis and to outline a plan to attract further independent funding, using the award to drive forward new competitively-funded research relevant to histiocytosis. The credibility of these sections of the application will be central to a successful outcome through the review process.
The scientific review board will meet to discuss applications in May and submit their recommendations to the Trustees for approval soon after.
Eligible costs include salaries and consumables for researchers directly engaged in the proposed study but will not include PI salary, overheads, IT equipment or travel.
Closing date for applications of 5pm GMT 18 February 2019.
Myotubular trust - 2019 call for projects (open to international applications)
Background
The Myotubular Trust (UK Registered Charity No: 1137177) was set up in 2006 to raise money for research to find a cure and / or treatment for any of the genetic forms of centronuclear and myotubular myopathy.
There are a number of genetically distinct forms of centronuclear myopathy. The commonest is x-linked, usually called myotubular myopathy, affecting in the vast majority of cases, only boys. It is also usually the most severe, presenting in the new-born period and there are associated breathing and swallowing difficulties in addition to the general muscle weakness. The other forms are either dominant or recessive in inheritance, are usually, but not always, milder, and vary widely.
2019 call for projects
Myotubular Trust is holding a 2019 call for research grants. We will require completed applications by 1700 hours GMT Friday 15th March 2019. We anticipate making awards in late June / early July.
We are looking to fund further projects that will help find a cure and / or a treatment for any form of centronuclear and myotubular myopathy (congenital X-linked recessive; congenital autosomal recessive; autosomal dominant), focusing on research that would not generally be funded by public or industrial funding sources. This call will be open to research bodies internationally.
We will be looking for the following types of application:
1. A project grant applied for by a Principal Investigator to fund a project for 2-3 years duration to be carried out by a Post-Doctoral researcher, or PHD student
2. A Myotubular Trust fellowship – basic science (3-4 years duration), where the scientist has identified a group that he or she wants to work with. Award is made to a named individual.
In particular, we would like to encourage the application of new technologies to research into centronuclear and myotubular myopathy; interventional trials; and those which may involve collaboration between different medical disciplines and / or different research institutions. We are also willing to consider applications which involve joint funding with other organisations.
Myotubular Trust’s Scientific Advisory Board (SAB) is chaired by Professor Francesco Muntoni of The Institute of Child Health, University College London. The SAB makes recommendations to the Myotubular Trust Trustees on which projects to fund, based on scientific assessment and peer review.
Further information
Further guidance and application forms can be found on the website.
Contact - research@myotubulartrust.org
Job opportunities
Scientific Officer within the European Virtual Research Platform for Rare Diseases
Please send your CV and cover letter to with the reference US14-2018-16 to:
- Ana RATH
- E-mail : jobs.orphanet@inserm.fr
- Tel : +33 (0)1 56 53 81 37
Mission manager - Rare diseases nomenclature
Please send your CV and cover letter to with the reference US14-2018-14 to:
- Annie OLRY
- E-mail : jobs.orphanet@inserm.fr
- Tel : +33 (0)1 56 53 81 37
Coordinator - Expert Services Directory
Please send your CV and cover letter to with the reference 2019-US14-002 to:
- Ana RATH
- E-mail : jobs.orphanet@inserm.fr
- Tel : +33 (0)1 56 53 81 37
Project Manager - The Institute of Myology
Located in Paris at the heart of the largest European hospital, Pitié-Salpêtrière, the Institute of Myology was created in 1996 by AFM-Telethon, a patient’s organization. Its goal: Promote Myology and have it accepted as a standalone clinical and scientific discipline. The Institute of Myology coordinates, around the patient, medical care, basic research, applied research, clinical research and teaching.
This fixed term contract, based at the Institute of Myology (Hôpital Pitié Salpêtrière, Paris 13) is to be filled on a full-time basis.
For further details consult the Myology website.
Thank you to send your application (CV + motivation letter) to the following address: recrutement-aim@institut-myologie.org
Project Assistant - Institute of Myology
Located in Paris at the heart of the largest European hospital, Pitié-Salpêtrière, the Institute of Myology was created in 1996 by AFM-Telethon, a patient’s organization. Its goal: Promote Myology and have it accepted as a standalone clinical and scientific discipline. The Institute of Myology coordinates, around the patient, medical care, basic research, applied research, clinical research and teaching.
This fixed term contract, based at the Institute of Myology (Hôpital Pitié Salpêtrière, Paris 13) is to be filled on a full-time basis.
For further details consult the Myology website.
Thank you to send your application (CV + motivation letter) to the following address: recrutement-aim@institut-myologie.org
Media
New app for economically deprived RD patients
A new application, FundFinder, has been developed by the Patient Access Network Foundation in order to help economically vulnerable patients with chronic or rare diseases.
Promising result for Cystic Fibrosis management app
The use of a mobile app, MyCyFAPP has helped some children affected by cystic fibrosis to manage their disease. According to the EU-funded project, it lessened the gastrointestinal symptoms and their worries of suffering pain.
New VASCERN video
Documentary video on Multiple System Atrophy Coalition
The documentary video Sophie's Search for a Cure, documenting the effects of Multiple System Atrophy (MSA) on Sophie and her family is now available on youtube.
Cure SMA resource
Cure SMA is a new resource which gives further details about Spinal Muscular Atrophy and videos about how to assist early diagnosis.