The future of Charcot-Marie-Tooth research and treatments rests where the disease begins: in genes. While many new genes involved in CMT have been identified, we know as many as 40 percent of people living with CMT still don’t have a confirmed genetic diagnosis. We’re also still trying to understand the disease mechanisms of the genes . . .
This is an unprecedented time for patients living with spinal muscular atrophy (SMA). It has now been almost two years since Spinraza became the first FDA-approved treatment for SMA, and in that time it’s proven to be a transformative therapy. Although there is much more work to be done, Spinraza has made a significant impact . . .