Sentences with phrase «called nusinersen»

An antisense - based treatment called nusinersen, which removes a roadblock in the expression of the second SMN gene, was recently approved by the FDA.

Yet a few parents have posted videos online showing children given the drug, called nusinersen, who appear to be sitting and even walking with assistance.

Not exact matches

Hua's extensive work in Krainer's lab, in collaboration with Ionis, led to the identification and preclinical development of nusinersen, a snippet of modified RNA called an antisense oligonucleotide that intervenes in the splicing process to ensure that SMN2 expresses the full - length SMN protein.