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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
Interview: Dr. Strober on Combination Therapy for Babies With SMA
Before 2016, there was no treatment to change the course of spinal muscular atrophy. Now, three t...
Posted 09/21/2021
Interview: Dr. Strober on Combination Therapy for Babies With SMA
Before 2016, there was no treatment to change the course of spinal muscular atrophy. Now, three t...
Posted 09/21/2021
Treatment With Evrysdi After Zolgensma Shows Benefit for SMA Type 1
Daily doses of risdiplam may provide benefits for children with type 1 spinal muscular atrophy af...
Posted 09/02/2021
Treatment With Evrysdi After Zolgensma Shows Benefit for SMA Type 1
Daily doses of risdiplam may provide benefits for children with type 1 spinal muscular atrophy af...
Posted 09/02/2021
Long-Term Data: Children With Type 1 SMA Keep Gaining Motor Skills After 3 Years on Evrysdi
Three innovative SMA treatments have emerged in recent years: risdiplam, nusinersen (Spinraza), a...
Posted 06/15/2022
Long-Term Data: Children With Type 1 SMA Keep Gaining Motor Skills After 3 Years on Evrysdi
Three innovative SMA treatments have emerged in recent years: risdiplam, nusinersen (Spinraza), a...
Posted 06/15/2022
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 ...
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...
New Trial Will Test Higher Dose of Nusinersen for People With Later-Onset SMA
A new worldwide clinical trial will test the safety and efficacy of higher doses of nusinersen, s...
Posted 10/05/2021
New Trial Will Test Higher Dose of Nusinersen for People With Later-Onset SMA
A new worldwide clinical trial will test the safety and efficacy of higher doses of nusinersen, s...
Posted 10/05/2021
Risdiplam Safe and Effective After 24 Months of Treatment in Babies With Type 1 SMA
On July 29, The New England Journal of Medicine published the results of a study led by Dr. Basil...
Posted 08/27/2021
Risdiplam Safe and Effective After 24 Months of Treatment in Babies With Type 1 SMA
On July 29, The New England Journal of Medicine published the results of a study led by Dr. Basil...
Posted 08/27/2021
SMA Medication Zolgensma May Cause Liver Failure, Warns New Label Update
New research suggests that Zolgensma — used to treat children with SMA — can cause liver damage a...
Posted 11/30/2021
SMA Medication Zolgensma May Cause Liver Failure, Warns New Label Update
New research suggests that Zolgensma — used to treat children with SMA — can cause liver damage a...
Posted 11/30/2021
What Is Kugelberg-Welander Disease?
Imagine a condition that gradually weakens the muscles you rely on every day to walk, run, and l...
What Is Kugelberg-Welander Disease?
Imagine a condition that gradually weakens the muscles you rely on every day to walk, run, and l...