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Decision-making and challenges within the evolving treatment algorithm in spinal muscular atrophy: a clinical perspective
Lakshmi Balaji
1
,
2 ,
Michelle A Farrar
1
,
2
,
3 ,
Arlene M D’Silva
1
,
2
,
3 ,
Didu S Kariyawasam
1
,
2
June 2023
There is no author summary for this article yet. Authors can add summaries to their articles on ScienceOpen to make them more accessible to a non-specialist audience.
Abstract
<p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" class="first" dir="auto"
id="d8848555e101">The clinical application of disease modifying therapies has dramatically
changed the
paradigm of the management of people with spinal muscular atrophy (SMA), from sole
reliance on symptomatic care directed toward the downstream consequences of muscle
weakness, to proactive intervention and even preventative care.
</p>
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Most cited references124
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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
W. Arnold, Louise Rodino‐Klapac, Thomas Prior … (2017)
- Record: found
- Abstract: found
- Article: not found
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
Richard Finkel, Eugenio Mercuri, Basil Darras … (2017)
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- Article: not found
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
Eugenio Mercuri, Basil Darras, Claudia A. Chiriboga … (2018)
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