The six things you need to know from the first day of CureDuchenne FUTURES

The first full day of FUTURES has wrapped! In the last couple of hours, we’ve had educational workshops on gene therapy, RNA-based therapeutics, and muscle restoration and anti-inflammatory treatments. Plus, everyone’s had a chance to meet a new person and create relationships that will last a lifetime. It’s been a lot to digest, so here are the top highlights from Friday at CureDuchenne FUTURES.

1. Gene therapies are on the way

We heard from biotech companies like Pfizer, Sarepta, Solid, REGENXBIO, Ultragenyx, and Vertex on the latest and greatest therapy that will help a wide array of boys, not just ones with certain mutations.

2. RNA-based approaches are evolving

In a two-part presentation, cutting-edge pharmaceutical companies outlined how their therapies are restoring dystrophin in muscle cells. Presenters included PTC, Sarepta, NS Pharma, Dyne, Entrada, PepGen, BioMarin, Avidity, and Wave.

3. New science is on the horizon

Creativity and science came together in the final science panel of the day as companies presented promising research that could change the future of Duchenne therapies. Myosana is using unique delivery vehicles to deliver full-length dystrophin into muscle cells, Code Bio created a synthetic DNA scaffolding to transport the dystrophin gene, Locanabio showed how snRNA can be used to increase the effectiveness of exon skipping, and Tevard Bio explained how synthetic tRNA could be the key to stop codon mutations.

4. CureDuchenne Link drives research forward

Our very own Heather Medlin, senior director of CureDuchenne Link, announced an exciting new partnership with PicnicHealth to make health data accessible while stressing the importance of collaboration to share bio specimens that can drive research forward.

5. The e-gaming lounge was a “Smash”

The moment we’ve all been waiting for finally arrived. LED walls, Super Smash Bros., flat-screens with colorful pixels flying lit up Pacific Ballroom rooms 24 through 26. There were plenty of smiles to go around as guys played together and sometimes against each other.

6. Duchenne Dads had some bonding time

Dads, uncles and grandpas tested out their golf game at the Topgolf Swing Suite. While hitting balls into a net was the main event, it also allowed dads some alone time to learn from each other and lean on the power of community to cope with a Duchenne diagnosis.