Sarah just got back from a caregiver advisory board meeting with the makers of Bennett’s infusion medication. We were celebrating the 10 year anniversary of the specific infusion drug that Bennett’s received weekly.
She was even interviewed to share their journey for a documentary.
And, Sarah learned a lot, there is currently a trial on a higher dose of the weekly infusion with data to hopefully be out later this year. They believe that the higher dose will prove to slow the progression of the disease at a higher rate.
She was also able to learn about the FDA approved gene therapy, see 3 year data from the therapy, and connect with families that have already received it.
All in all, it was a great two days with lots of information and connecting with families and others in the RARE community.
We will be discussing the possibility of gene therapy in combination with exon skipping for Bennett once we get back from the FUTURES conference in Orlando later this month. Thats right, Bennett will be attending his first Cure Duchenne conference in just a couple of weeks.
Stay tuned for more news and info about whats to come and what is on the forefront for DMD warriors like Bennett!
