- Nusinersen,
marketed as
Spinraza, is a
medication used in
treating spinal muscular atrophy (SMA), a rare
neuromuscular disorder. In
December 2016, it became...
-
plans on
developing its Hemophilia-focused goals. In 2016,
Biogen released Spinraza (nusinersin), a
treatment for
Spinal Muscular Atrophy. The drug is among...
- RNA-targeted therapeutics. The
company has
three commercially approved medicines:
Spinraza (Nusinersen),
Tegsedi (Inotersen), and
Waylivra (Volanesorsen) and has...
- PMID 32006461. "
Spinraza (nusinersen) Injection". U.S. Food and Drug
Administration (FDA). 18
January 2017.
Retrieved 8
August 2020. "
Spinraza EPAR". European...
- FDA-approved drug,
known as
Spinraza was
developed by Dr.
Adrian Krainer and his Cold
Spring Harbor Laboratory colleagues.
Spinraza is an
oligonucleotide that...
- for treatment,
sometimes as high as $700,000 a year, as in the case of
Spinraza (Biogen), FDA
approved in
December 2016 for
spinal muscular atrophy, placing...
- of
oligonucleotides to the brain,
including the
recent FDA
approval of
Spinraza. Wahlestedt's team, with collaborators,
introduced the now
widespread use...
-
Exondys 51
Duchenne muscular dystrophy Sarepta Therapeutics Rare
pediatric Spinraza Spinal muscular atrophy Ionis Pharmaceuticals Rare
pediatric Emflaza Duchenne...
-
medical interventions that
target the RNA-splicing process,
including Spinraza,
which is the
first treatment for the
genetic disorder Spinal Muscular...
-
targeting therapeutics accessible throughout the world, she
chairs the
Spinraza Individual Patient Humanitarian Access Program Medical Expert Committee...
