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Top 10 search results for "the importance of splicing in sma treatment" in Resources. To see all results and access other features, sign up for free.

Spinal muscular atrophy (SMA) is a genetic disease that destroys nerve cells in the spinal cord a...

The Importance of Splicing in SMA Treatment

Spinal muscular atrophy (SMA) is a genetic disease that destroys nerve cells in the spinal cord a...
The researchers who discovered and developed the spinal muscular atrophy (SMA) treatment Evrysdi...
Posted 03/09/2022

Evrysdi Development Team Wins Drug Discovery Award for Oral SMA Treatment

The researchers who discovered and developed the spinal muscular atrophy (SMA) treatment Evrysdi...
Posted 03/09/2022
When a child is born with spinal muscular atrophy (SMA), a rare neuromuscular disorder, it’s the ...

Understanding How SMA Gene Therapy Works

When a child is born with spinal muscular atrophy (SMA), a rare neuromuscular disorder, it’s the ...
Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder characterized by muscle weaknes...

Causes of Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder characterized by muscle weaknes...
New data shows that risdiplam (sold as Evrysdi) helps most infants with spinal muscular atrophy (...
Posted 06/15/2022

Long-Term Data: Children With Type 1 SMA Keep Gaining Motor Skills After 3 Years on Evrysdi

New data shows that risdiplam (sold as Evrysdi) helps most infants with spinal muscular atrophy (...
Posted 06/15/2022
Spinal muscular atrophy (SMA) type 1 is the most common type of spinal muscular atrophy. SMA is a...

Spinal Muscular Atrophy Type 1

Spinal muscular atrophy (SMA) type 1 is the most common type of spinal muscular atrophy. SMA is a...
Clinical trials may test new drugs or new dosages or combinations of drugs for spinal muscular at...

5 Reasons To Consider Participating in SMA Clinical Trials

Clinical trials may test new drugs or new dosages or combinations of drugs for spinal muscular at...
Corrective surgery for scoliosis in children with spinal muscular atrophy (SMA) type 1 can lead t...
Posted 01/18/2022

Scoliosis Surgery in Kids With SMA Can Cause Serious Complications, Research Finds

Corrective surgery for scoliosis in children with spinal muscular atrophy (SMA) type 1 can lead t...
Posted 01/18/2022
Spinal muscular atrophy (SMA) type 2 is a genetic condition that causes muscle weakness (hypotoni...

Spinal Muscular Atrophy Type 2

Spinal muscular atrophy (SMA) type 2 is a genetic condition that causes muscle weakness (hypotoni...
Finkel type SMA (SMAFK) is a rare type of adult-onset (or late-onset) spinal muscular atrophy (SM...

What Is Finkel Type SMA?

Finkel type SMA (SMAFK) is a rare type of adult-onset (or late-onset) spinal muscular atrophy (SM...