Nusinersen, marketed as Spinraza, is a medication used in treating spinal muscular atrophy (SMA), a rare neuromuscular disorder. In December 2016, it became...

outcomes. Medications that target the genetic cause of the disease include nusinersen, risdiplam, and the gene therapy medication onasemnogene abeparvovec....

therapeutics. The company has three commercially approved medicines: Spinraza (Nusinersen), Tegsedi (Inotersen), and Waylivra (Volanesorsen), and has four drugs...

increase in the concentration of the functional SMN protein in vivo. Nusinersen, the first drug approved to treat SMA, an anti-sense oligonucleotide targeting...

oligonucleotide later named nusinersen was developed by Ionis Pharmaceuticals under a licensing agreement with Biogen. In December 2016, nusinersen received regulatory...

Laboratory in Laurel Hollow, New York for the early-stage development of nusinersen. Nusinersen became FDA approved in 2016 as the first treatment for spinal muscular...

inflammatory bowel disease 2019 2018 Biosimilar of AbbVie's Humira. SPINRAZA (nusinersen) Spinal muscular atrophy (SMA) 2016 2017 The first drug approved for the...

and National Academy of Sciences member. Adrian R. Krainer, developed nusinersen to treat spinal muscular atrophy (SMA), winner of the Breakthrough Prize...

stage 4 gallbladder cancer. Later, this cancer spreads to her brain! Nusinersen is a medication used in treating spinal muscular atrophy (SMA). Jesse...

N1 (ISS-N1) target that led to the first FDA-approved drug (Spinraza/Nusinersen) for the treatment of spinal muscular atrophy (SMA). He is the recipient...