As more and more people affected by SMA gain access to the recently approved Spinraza therapy, Cure SMA has released its pipeline that looks at how research is going in other areas that may benefit the spinal muscular atrophy (SMA) community.
According to the organization, there are currently 19 SMA therapies in clinical trials, and two of them are nearing the end of their phase II trials — AVXS-101 and Olesoxime. Cure SMA’s research program is working with 14 different pharmaceutical companies to develop better gene therapies and drug treatments to help improve the lives of those living with the disease.
Cure SMA is funding research into two main areas: SMN-based or SMN-enhancing therapies which aim to boost the body’s ability to produce survival motor neurons (SMN) and non-SMN therapies which look to improve the function of muscles and nerves.
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