Record Number of Abstracts Submitted for 2018 SMA Researcher Meeting

Abstract submission is now closed for the 2018 SMA Researcher Meeting. We are excited to announce that we received 128 submissions, a record number. Our submissions come from researchers in 16 countries, 11 companies, and close to 50 institutions.

We expect over 400 researchers and clinicians from almost 100 organizations will attend the SMA Researcher Meeting. With registration for both the family and researcher portions of the Annual SMA Conference on track to reach capacity, we anticipate an engaging and productive conference.

Next Steps

Abstract submissions allow scientists from all over the world to submit new and unpublished data for consideration. From the submissions received, our Scientific Advisory Board will select the most captivating and promising to be presented at the conference. While considering abstract submissions, the SAB will also look for areas of overlap or commonality. Often, different scientists working on similar questions can be gathered into a single session to stimulate new discussions and collaborations that can further SMA research.

One of the most important themed sessions each year is our special session. This year’s special session is called, ‘Clinical and Basic Questions of SMA in the Era of SMN Repletion: Challenge and Opportunity’. As we celebrate the long awaited first drug approval, we know there remains much to be done. While Spinraza offers tremendous benefit to those with SMA, there is also a need for continued research. The development of additional therapies to be used independently or in combination with SMN enhancing approaches requires understanding more about SMA biology and disease mechanisms. Studying the “new” SMA phenotype after treatment will unveil new, unmet patient need, perhaps including extra-neuronal phenotypes and the need for such therapies. The goal of this session is to discuss these new challenges and opportunities.

The session begins by exploring the efficacy and unmet needs of current treatment and is followed by investigating patient phenotypes post treatment. Next, the neurodevelopmental need for prenatal SMN is discussed and its requirement for complete restoration of phenotype. Following that will be a discussion of the unanswered basic biology questions pertaining to SMA and their implications for therapy development. The panel discussion will focus on prioritizing future research endeavors to address unmet medical need for SMA patients. The moderator for the special session is Richard Finkel, MD, Chief, Division of Neurology, Nemours Children’s Hospital.

The SAB also extends invitations to other scientists working on experiments that might be helpful to the SMA research community. In the past, this has included researchers working on related diseases like ALS, in order to see what we can learn from advancements in other neuromuscular disorders.

How We Are Engaging the SMA Community

Along with our open requests for funding proposals, open abstract submission allows us to engage the SMA researcher community and accelerate the pace of research. In addition, many of the scientists who submit abstracts or who are invited to give talks will also be invited to prepare posters for our family-friendly poster session, during which families can interact directly with researchers and ask them questions about their work.

A panel discussion on the drugs in various stages of the SMA drug pipeline is also planned, and will be open to families attending the conference. The preliminary agenda for the 2018 SMA Researcher Meeting can be found here.

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