International conference: Paving the road to therapy

Dear Lama2-families,
We are organizing an international conference for the Lama2 community together with professor Bert Smeets from Maastricht university. The conference is entitled “LAMA2 Muscular Dystrophy: Paving the road to therapy” and we are able to organize it thanks to funding of the EJP RD Networking Support Scheme round 7. This conference will take place in Barcelona from Friday 17th untill Sunday 19th of March. On Saturday 18 march you are welcome to join the online programme, specially made for patients and families.

The first aim of the Networking Support Scheme in the European Joint Programme on Rare Diseases (EJP RD) is to encourage sharing of knowledge on rare diseases between health care professionals, researchers, and patients/ patient advocacy organisations in new or expanding research networks by funding networking events.

The second aim of the Networking Support Scheme is to enable or increase the participation of usually underrepresented countries in Europe in new and in expanding research networks on rare diseases.

The focus of the workshops or conferences should be (the implications of) research results and innovative solutions. The results of these networking events may lead to future collaborative and novel research efforts.

Our aim is to meet all these objectives at this three-day conference.

Lama2 Muscular Dystrophy: Paving the road to therapy
The organizing consortium of the networking event consists of the main clinical and research groups and patient organizations in the EU, but key participants are invited from all over the world. The outcomes will be disseminated to all European and affiliated countries.

The conference
The first and last day of the 3-day event are by invitation only. We will focus on patient registries, ongoing natural history studies to align them and define biomarkers and guidelines which can be used in patient-care and defining diagnostic criteria and guidelines for alleviating symptoms and maintaining quality of life. These will be distributed among patients, patient organizations and clinicians throughout Europe. We also focus on therapy development for LAMA2-MD by making an inventory of available patient samples for research, sharing confidential research progress and establishing a LAMA2-MD therapy task-force.

The last day we want reach a conclusion/consensus on the natural history studies, treatment strategies and patient requirements. This should lead to a concrete action plan. After the meeting we intend to publish a meeting report.

Online and open for all patients
So, the idea is to have two days for researchers and clinicians and one day for patients and families. We want to involve the patients as much as possible, because they are the reason we are doing all of this. The second day we want to give some general lectures about several important topics for patients and families. The lectures will be given in English and will be comprehensible to laymen.

After the lectures, we give the patients and their families (and possibly other interested parties such as clinicians and physiotherapists) the opportunity to ask questions in an online meeting with various breakout sessions per theme. Experts (clinicians, researchers and representatives from patient organisations) will be there to answer all the questions.

We are currently working on a website with the programma and all the information about the conference. If you have any questions, please fill in our contact form and we will try to answer you. If you want to join, please keep an eye on this website for more information!