Several key factors influence how and why some patients and their families may be hesitant to pursue, or decide to forego, Spinraza (nusinersen) treatment, a study has found. These considerations ... Read more
A population-based study conducted in the state of New York demonstrates the feasibility and benefits of newborn screening for spinal muscular atrophy (SMA). Supported by these findings, researchers recommend adding ... Read more
Targeted therapies or dietary supplements that work to improve the metabolism of certain molecules in muscle cells may slow the progression of spinal muscular atrophy (SMA), results of an ... Read more
Spinraza. This word elicits a different emotion for each person. Much like SMA itself, everyone’s experience with this medical treatment is different. For some, it has been life-changing in a ... Read more
SMA experts and patient representatives concede they have a long way to go in updating a consensus document on SMA standards of care.
Their report, which recently appeared in the journal Neuromuscular ... Read more
Subcutaneous administration of a new class of antisense oligonucleotide (ASO) compounds called tricyclo-DNA may be an efficient therapy for spinal muscular atrophy (SMA), according to a study in mice. ... Read more
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