SMA Europe Reports on its Activities

16 April 2019

SMA members met in Bucharest on 4th - 6th April and reported as follows:

New members

Finland and Belgium have joined our European consoritum, both as observers in the first instance. After one year, they will have the choice of joining as full or associate members. SMA Europe now comprises of 19 countries.

Congress 2020

The organisation of the second international scientific and clinical congress is in full swing. It will take place between 5th and 7th February 2020 in Paris. The programme will include sessions on fundamental research, clinical trial developments and industry symposia, as well as an opening speech by Professor Melki, who led the team who discovered the gene responsible for SMA, in Paris, in 1995.

Call for Research Project Proposals

The call for research proposals was launched in February 2019 and attracted 20 applications. These are currently being peer-reviewed and funding decisions will be made at SMA Europe's next meeting in October 2019.

Other projects are also on-going and progressing well. These include:

  • A mapping exercise of SpinrazaTM access across Europe with the aim of sharing experiences to inform possible approaches to national access and reimbursement
  • Participating in the international group tasked with translating the revised Standards of Care into a family-friendly version
  • Advocating for inclusion of the patient voice in drug development
  • The development of an advocacy tool to inform drug development and approval. 

We will report on these in due course.

To find out more about SMA Europe, please visit their website