“Our findings suggest that treating the whole body, not just the nerves, could improve the health, quality of life and survival of people with SMA.”- Dr. Rashmi Kothary
Treating a patient’s entire body, not just the nerves in their muscles, is essential for the complete treatment of spinal muscular atrophy (SMA), according to a study led by Aoife Reilly in Dr. Rashmi Kothary’s group.
Patients with SMA have debilitating muscle weakness caused by depletion of the SMN protein, and their heart, liver, pancreas, immune system and digestive systems may also be affected. Thankfully, new therapies that restore the SMN gene can dramatically improve their outcomes. However, it’s unclear which delivery route is best; through the blood stream or directly through the spinal cord.
(as reported by the OHRI on July 13, 2022)
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