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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 Importance of Splicing in SMA Treatment
Spinal muscular atrophy (SMA) is a genetic disease that destroys nerve cells in the spinal cord a...
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
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
Understanding How SMA Gene Therapy Works
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 ...
Causes of Spinal Muscular Atrophy
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...
Spinal Muscular Atrophy Type 1
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...
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
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
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...
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...
What Is Finkel Type SMA?
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...
Understanding Spinal-Bulbar Muscular Atrophy (SBMA)
Spinal muscular atrophy (SMA) is a motor neuron disease caused by genetic mutations passed on fro...
Understanding Spinal-Bulbar Muscular Atrophy (SBMA)
Spinal muscular atrophy (SMA) is a motor neuron disease caused by genetic mutations passed on fro...
Spinal Muscular Atrophy Type 2
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...