| Nn101 originated from the ohio state university wexner medical center and nationwide children’s hospital in columbus, oh. | Enrollment of all healthy control infants was completed in less than one year. |
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| Spinal muscular atrophy type 1 sma1 is the leading genetic cause of infant mortality for which therapies, including avxs101 onasemnogene abeparvovec, zolgensma® gene replacement therapy, are emerging. | It was sponsored by ninds u01 ns079163, curesma, the muscular dystrophy associate, sma foundation, families of sma, and wrybaby. |
| Home projects nn101 sma spinal muscular atrophy sma biomarkers in the immediate postnatal period of development spinal muscular atrophy sma is a rare, inherited disease that results in loss of nerves in the spinal cord and weakness of the muscles connected with those nerves. | Enrollment began in november, 2012 and ended september, 2014. |
Spinal Muscular Atrophy Type 1 Sma1 Is The Leading Genetic Cause Of Infant Mortality For Which Therapies, Including Avxs101 Onasemnogene Abeparvovec, Zolgensma® Gene Replacement Therapy, Are Emerging.
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