Euro-ataxia

Last week Euro-ataxia hosted 18 ataxia patient organisations for the International Patient Conference. We heard updates on clinical trials, and talked about our experiences of the drugs regulatory process, amongst other topics! Keep a look out for a summary of the event coming soon on our website!

Gene therapy survey

You are invited to participate in an online research survey, about gene therapy for genetic ataxias. The purpose of this survey is to collect opinions about gene therapy research from people diagnosed with a genetic ataxia from a number of countries.

Gene therapy is a potential treatment for genetic ataxias, and as such the views of people with the condition are invaluable in the design of future research and clinical trials. The results of this research will provide information to the ataxia research community about opinions on gene therapy, which will help when designing future treatment trials.

People diagnosed with a genetic ataxia, or their family members, are eligible to complete the survey. Minors are required to complete the survey with a parent or guardian. We expect the survey will take roughly 30 minutes to complete, and the data collected will be anonymous.

If you have any questions about this research, contact Ataxia UK’s research department at [email protected].

Gene Therapy Survey for People with Genetic Ataxias Take this survey powered by surveymonkey.com. Create your own surveys for free.

https://investors.biogen.com/news-releases/news-release-details/biogen-received-european-commission-approval-skyclarysr

With the comment from our President Andreas Nadke.

“On behalf of Euro-ataxia and its members, I am delighted to welcome the European Commission approval of SKYCLARYS, a much-needed medicine for adults with Friedreich’s ataxia in EU countries,” said Andreas Nadke, President of Euro-ataxia. “Our member’s patient groups have been working and waiting for this day for many years, and we firmly believe that this will be a successful and gratifying beginning in the treatment of Friedreich’s ataxia.”

Biogen Received European Commission Approval for SKYCLARYS® (omaveloxolone), the First Therapy to Treat Friedreich’s Ataxia | Biogen The Investor Relations website contains information about Biogen's business for stockholders, potential investors, and financial analysts.

Breaking News!

Ataxia UK and CureDRPLA have launched the CureDRPLA Global Patient Registry, the only existing registry of Dentatorubral-pallidoluysian atrophy (DRPLA) patients. Having a patient registry is essential for collecting information to better understand DRPLA development and monitoring the quality of health. This registry is designed to house patient-entered data; when joining the registry, participants will be asked to answer questions about the DRPLA diagnosis, medical history, activities of daily living, etc. Participation is available to all DRPLA patients and families regardless of country of residence and the questionnaire is available in English, French, Italian, Portuguese, Japanese and Korean.

To find out more and participate please visit:

CureDRPLA Global Patient Registry | CureDRPLA The goal of the CureDRPLA Global Patient Registry is to collect information on all DRPLA patients in one registry. By collecting the information on all DRPLA patients, the CureDRPLA Global Patient Registry will be a powerful resource for research and will enhance opportunities for treatment developm...

Join our virtual research conference on Friday 18th June 14-17:30 CET. As part of the Euro-ataxia annual meeting we have arranged for ataxia researchers from universities and pharma companies to talk to us about recent research developments. Please register here: https://us02web.zoom.us/meeting/register/tZcsd-mtrz0oHdU1DddlrTmmCj2HsVtsHxCK and see the programme here: https://www.euroataxia.org/research-overview/

Research Overview – EuroAtaxia Research Overview One of the main aims of Euro-ataxia is to accelerate research and treatments for people with ataxia. This co-ordinated effort between our member organisations supports research in a number of ways, such as providing patient input and advice to research studies and assisting in recr...

Tomorrow it's Rare Disease Day!

Rare disease day was created by EURORDIS - European Rare Diseases Organisation and its Council of National Alliances and takes place every year across the world on the last day of February, a “rare date”. Its aim is to raise awareness of rare diseases and the impact it has on patients’ lives amongst the general public and decision makers.

The theme for rare disease day is
- Rare is many worldwide
- Rare is strong every day
- Rare is proud everywhere’.

Why not show your support by putting up a poster where you work, sharing your stories or sharing our posts across our social media channels to help to raise awareness of rare diseases?

Visit the event page on our website for more info on how you can get involved: https://bit.ly/2VBTRhM or go to the rare disease day website www.rarediseaseday.org/

The next EURORDIS Summer School on Medicines Research & Development will take place on 8-12 June 2020 in Barcelona. The EURORDIS Summer School provides rare disease patient advocates and researchers working with rare diseases with the knowledge and skills needed to become experts in medicines R&D. Apply by 30 November:

EURORDIS Summer School 2020 - Application form Carefully read all the information below before applying. The application form will appear once you click “next” at the end of this page.

Have you been involved in biomedical research projects? If so, EURODIS would like to hear from you! They are looking for patient representatives to join a new working group to describe the role and expectations of patient involvement in research proposals, to provide guidance to applicants and develop guidelines.

These guidelines will support fruitful partnerships between scientists and patient organisations, paving the way for systematic patient-centered research. More information on how to apply here: https://bit.ly/3392QIf (deadline 11 November) or contact [email protected] with any questions.

download2.eurordis.org.s3.amazonaws.com

Rare2030, the EURORDIS-led foresight study on the future of rare disease policy, has now brought together nearly 200 key opinion leaders in rare disease policy in a Panel of Experts, which will meet in person for the first time in Brussels on 7 November to propose ideal future policy scenarios in the fields of rare disease diagnostics, treatment, care and social support.

Join the opening session of the Brussels conference online on 7 November and share your comments at: https://www.rare2030.eu/live/

Any questions please contact [email protected].

Live streaming The health of 30 million people living with a rare disease in Europe should not be left to luck or chance. Rare 2030 prepares a better future for people living with a rare disease in Europe with foresight. 1 Watch online Live stream Rare2030 Panel of Experts Workshop 9:00 – 11:30 Brussels time, 7 ...

The two ERNs for Rare Neurological Diseases and Rare Neuromuscular Diseases (Ern-Rnd European Reference Network for Rare Neurological Diseases and ERN-EuroNMD) are launching a new joint educational program in collaboration with the European Academy of Neurology.

The goal is to share knowledge on rare neurological, movement and neuromuscular disorders and fill the gap in the educational offer in these fields. Our target audiences are clinical specialists (neurologists and pediatric neurologists), neurology trainees. And other non-medical staff involved in the care of neurology patients.

The webinars will treat different aspects of the disease groups covered in their networks such as clinical features, disease diagnosis and management, use of scales and flowcharts, and possible therapeutic interventions. Topics relevant for adult and paediatric cases will have equal share in the curriculum.

In order to make the webinars interactive and ensure that the learning objectives are met they will integrate polling questions.

The first webinar will be on Clinical features of ataxia and will be presented by Bart van de Warrenburg from the Radboud University Medical Center in Nijmegen. It will take place on 5 November from 15-16h CET.

To register you can click here: https://zoom.us/webinar/register/3515699249976/WN_Ydd3V8KJSR-zaj2HpkSAKw

For further information and to register for the webinars please visit: http://www.ern-rnd.eu/education-training/webinars/

Welcome! You are invited to join a webinar: Clinical Features Of Ataxia by Bart van de Warrenburg. After registering, you will receive a confirmation email about joining the webinar. Joint webinar series by the European Reference Networks for Rare Neurological Diseases (ERN-RND) and Neuromuscular Diseases (ERN-EuroNMD) and the European Academy of Neurology (EAN). The speaker is neurologist from the Radboud University Medical Centre in Nijmegen, the Netherlands. He is an expert o...

New Brain, Mind and Pain Book of Evidence released for the 2019-2024 EU mandate

The Brain, Mind and Pain (BMP) initiative yesterday launched its Book of Evidence for the 2019-2024 EU mandate: www.brainmindpain.eu

This Book of Evidence (BoE) sets a strategic vision on policy action to deliver better care for people with neurological and chronic pain disorders in the EU.

It will be used as the core policy document of the MEP Interest Group on Brain, Mind & Pain – which will continue after the upcoming EU elections for the next 5 year mandate.

The Interest Group is coordinated by European Federation of Neurological Associations (EFNA) and Pain Alliance Europe (PAE).

The BoE outlines clear focus areas and proposes recommendations for policy actions, which, if implemented, would significantly improve quality of life for people with neurological and chronic pain disorders, as well as their families and carers.

The development of the document was led by EFNA and PAE and involved a wide range of key EU and national stakeholders from the patient, policy, industry and scientific communities.

This collaborative development process is reflected in wide ranging endorsements and support from organisations such as the European Commission, European Brain Council, European Patients’ Forum, European Academy of Neurology, European Pain Federation and European Alliance for Patient Access.

Key areas for action

The BoE outlines the thematic areas of focus for the MEP Interest Group on Brain, Mind and Pain for the 2019-2024 mandate. These are:

» Eradicate stigma, isolation and discrimination: A lack of public awareness of BMP disorders feeds their stigma and the associated isolation and discrimination suffered by BMP patients, carers, and their families

» Ensure equitable access to treatment, services, and support: Access to treatment, services, and support is a topic of concern for patients in Europe due to high failure rates, delays in market access, relatively expensive treatments, and inequality in access across, but also within, EU Member States

» Promote patient empowerment for increased involvement and engagement: Patient empowerment is a means for more effective BMP patient engagement and meaningful involvement in the research, medical and policy conversations which affect them

These areas have been chosen in order to support the MEP Interest Group in leveraging current policy opportunities on the agenda whilst ensuring continuity with the past activities and successes of the BMP initiative.


Driving policy change

The 2019-2024 mandate brings with it an active policy environment at EU level, with numerous opportunities for advocacy on behalf of people with brain, mind and pain disorders.

Ensuring that the Interest Group is capable of impacting policy discussions throughout the 2019-2024 mandate will be key in making progress in the three key thematic areas identified.

Additionally, with the global priority turning more and more towards NCDs, we need to ensure that brain, mind and pain disorders are part of global health policy agenda.

To this end, each chapter of the BoE contains a section on the policy opportunities and subsequent actions that can be leveraged to further progress in each thematic area and at EU and global level.



Looking ahead

It is now the responsibility of the Interest Group and the wider BMP initiative to leverage the policy opportunities and pursue the policy actions identified in this text throughout the 2019-2024 mandate.

EFNA President, Joke Jaarsma says: ‘Progress is contingent on coordinated policy advocacy at EU and global level, taken forward by the BMP initiative as a whole and its partners.

‘Using the BoE as a springboard for action, let’s drive policy change and make a real difference to the daily lives of people living with BMP disorders across Europe!’ she continued.

For any further information on the BoE or the activities of the BMP initiative – including its MEP Interest Group – please do not hesitate to contact EFNA at: [email protected]

Brain, Mind and Pain - Find us on social media and tag your posts #brainmindpain Event reports – 2014-2019 mandate Fighting Discrimination at Work for those affected by neurological disorders and chronic pain conditions March 6th, 2019, European Parliament, Brussels Host MEP: Jeroen Lenaers. Click here to read the meeting report. Future of Healthcare in Europe What next for br...