Duchenne MD Drug Could Be Effective in Fighting SMA

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by Wendy Henderson |

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In this video from Wochit News, we find out a little information about a drug which has shown promise in mouse models in regressing spinal muscular atrophy (SMA).

Clinical development for SMA treatments reviewed at an annual conference. Find out more. 

The drug Pip6a-PMO, is designed for patients with Duchenne muscular dystrophy but showed promise in disease regression in mice with SMA. The researchers will be undertaking clinical trials in patients affected by SMA next year and hope that the drug will be successful and can be used for other similar diseases. Find out more about this drug and the proposed new delivery system for the treatment here.

Update on stage 2 clinical trial of nusinersen in SMA infants presented by Ionis Pharmaceuticals.

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