Spinal muscular atrophy (SMA) is a rare genetic condition that impairs a person’s ability to control their muscle movement. Most types of SMA are diagnosed in infants, but the condition sometimes ...
Peter L. Salgo, MD: Now we’re going to look at something called spinal muscular atrophy [SMA]. I must tell you that before researching it for this broadcast, it was not a disease that was on my radar.
The cost burden of patients with SMA is considerable, and is estimated to be approximately $4 million to $5 million over 10 years in patients with early-onset SMA. This cost is 54.2 times greater than ...
"The availability of disease-modifying therapies for patients with other types of SMA suggests that these key disease characteristics in SMA 3 patients should be amenable to therapy." — Barry J. Byrne ...
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 ...
Spinraza, also known as nusinersen, is a medicine used to treat spinal muscular atrophy (SMA) in children and adults. SMA is a genetic disease that is passed down through families. It leads to muscle ...
Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...
"Decreasing AMMO [active maximal mouth opening] over time is seen most often in patients with SMA type 2 with an estimated reduction of almost 1 mm per year...At this point, we propose that ...
We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Topline results from a phase 3 clinical trial showed an ...
Early diagnosis and treatment of SMA can limit disease progression in children and adults, extending life expectancy and improving QOL. Spinal muscular atrophy (SMA) is a severe genetic condition that ...
Abu Dhabi has made medical history after Sheikh Khalifa Medical City (SKMC) became the first in the world to administer a ...
Evrysdi, also known as risdiplam, is an oral medicine used to treat spinal muscular atrophy (SMA) in children and adults. SMA is a genetic disease that is passed down through families. It leads to ...