Annual SMA Conference

This June, the BfS Board Member Natalia Miller and the Executive Director Sage Hancock attended the 2024 Annual SMA Conference in Austin, Texas – to join over 2,260 attendees from over 28 countries working together to try and cure SMA! 

The Conference featured 68 thematic workshops held to share knowledge and network among more than 600 health care professionals in attendance. The meetings furthered research by building productive cross-disciplinary collaborations between new researchers, clinicians and drug companies. Overall, the Cure SMA community has had an incredible success with fundraising and crowdfunding over years. In Sage’s words: “This makes me want to see how we can do more!”

In total, 15 million has been invested into basic research about SMA, 20 million into drug discovery, and 3 FDA approved treatments have come from this funding! Lots of new treatments are in phase 2 and 3 right now. Several trials are also to further the work behind three current medications looking to improve them.

Special sessions were devoted to patients with SMA, their personal experiences. Aimed at better understanding the unmet needs, the panel discussions covered the burning issues from air travel to reproductive health, from the impact of anesthesia to frustrations due to lack of expertise with SMA in the field of physical and occupational therapy.

The common opinion is that the Standard of care for SMA needs to be adjusted since it was written before the disease modifying treatment was created. For instance, no special diets are needed with SMA, while yoga can be incredibly beneficial for patients of all ages. The benefits of dual therapy were also briefly discussed, since some patients have had success first with gene therapy then Risdiplam.

To sum up, this was an impactful and inspiring weekend of opportunities to connect, learn, and have fun, and our team is already looking forward to the 2025 Conference in Anaheim, California.

See you there!