Nearly all children with spinal muscular atrophy who started on Evrysdi before symptom onset are walking after three years, ...
"Our preliminary findings show that German children with SMA, despite significant physical disability, have surprisingly good subjective HRQoL." – Erik Landfeldt, MSc, PhD Previous studies have found ...
Biogen has pointed to an early readout from a small phase 1 trial as evidence that its antisense oligonucleotide salanersen could help children with spinal muscular atrophy (SMA) who are still ...
—Children with neuromuscular diseases and cerebral palsy often report disrupted sleep, which is usually attributed to breathing disorders. But new research points to a new potential contributor: ...
Please provide your email address to receive an email when new articles are posted on . At the time of last follow up, 82.6% of children born with SMA type 1 from 2016 on were still alive. Of the 140 ...
Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new 5-year data confirming the sustained efficacy and safety profile ...
The STEER study showed significant motor function improvement in children with SMA using intrathecal onasemnogene abeparvovec. The primary efficacy endpoint was met with a 2.39-point improvement in ...
Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...
In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene ...
Panaji: As Ruby Borges accompanies her nine-year-old son Dylan into and out of the intensive care unit (ICU) and watches his condition gradually deteriorate, she pins all her hope on state govt. Dylan ...
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