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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
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More Than Half of People Who Start Spinraza for SMA Stop Within 2 Years, Study Finds

People who start taking Spinraza (nusinersen) to treat spinal muscular atrophy (SMA) may not alwa...
Posted 12/15/2021

More Than Half of People Who Start Spinraza for SMA Stop Within 2 Years, Study Finds

People who start taking Spinraza (nusinersen) to treat spinal muscular atrophy (SMA) may not alwa...
Posted 12/15/2021
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Nasal Regurgitation With Spinal Muscular Atrophy

Nasal regurgitation — instances when food or fluids you’ve swallowed come up through your nose — ...

Nasal Regurgitation With Spinal Muscular Atrophy

Nasal regurgitation — instances when food or fluids you’ve swallowed come up through your nose — ...
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The Role of SMN1 and SMN2 Genes in Spinal Muscular Atrophy

Parents of children with spinal muscular atrophy (SMA) must learn a lot very quickly. Genetics is...

The Role of SMN1 and SMN2 Genes in Spinal Muscular Atrophy

Parents of children with spinal muscular atrophy (SMA) must learn a lot very quickly. Genetics is...
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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...
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Participants Sought for Clinical Trial of SMA Muscle Therapy Apitegromab

Pharmaceutical company Scholar Rock is seeking participants for a clinical trial of apitegromab, ...
Posted 12/22/2021

Participants Sought for Clinical Trial of SMA Muscle Therapy Apitegromab

Pharmaceutical company Scholar Rock is seeking participants for a clinical trial of apitegromab, ...
Posted 12/22/2021
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SMA Treatment for Children vs. Adults: 5 Key Differences

Spinal muscular atrophy (SMA) mainly affects children, but it doesn’t spare adults — 35 percent o...

SMA Treatment for Children vs. Adults: 5 Key Differences

Spinal muscular atrophy (SMA) mainly affects children, but it doesn’t spare adults — 35 percent o...
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Spinraza Alone May Be Better Than Combining It With Zolgensma To Treat SMA Type 1

A recent study has found that combining Zolgensma and Spinraza does not improve motor or respirat...
Posted 02/23/2022

Spinraza Alone May Be Better Than Combining It With Zolgensma To Treat SMA Type 1

A recent study has found that combining Zolgensma and Spinraza does not improve motor or respirat...
Posted 02/23/2022
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What Is Kugelberg-Welander Disease?

Imagine a condition that gradually weakens the muscles you rely on every day to walk, run, and li...

What Is Kugelberg-Welander Disease?

Imagine a condition that gradually weakens the muscles you rely on every day to walk, run, and li...
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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 ...
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