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 ...
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 ...
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 ...
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 customerservice@slackinc.com. Topline results from a phase 3 clinical trial showed an ...
Abu Dhabi has made medical history after Sheikh Khalifa Medical City (SKMC) became the first in the world to administer a ...
Treating Type 1 Spinal Muscular Atrophy: Spinal muscular atrophy is a rare, progressive neuromuscular disease. Type 1 SMA is characterized by the inability to sit without support and by reduced life ...
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 ...