Trusted Resources: Education
Scientific literature and patient education texts
Severity of SMA Muscle Impairment Prior to Treatment May Have Implications for Nusinersen Efficacy
source: The American Journal of Managed Care - AJMC
year: 2022
authors: Jaime Rosenberg
summary/abstract:A new study has found that the severity of muscle impairment and scoliosis before treatment may help predict response to nusinersen for certain patients with spinal muscular atrophy (SMA).
The retrospective study of patients with SMA type 2 and 3, published in Brain and Development, found that the severity of fatty infiltration and muscle atrophy at the start of treatment had implications for changes in motor function after treatment with nusinersen. The findings, which the researchers say highlight the importance of early diagnosis and initiation of treatment, come amid efforts to determine efficacy trends among the several treatment options for the condition.
read more
Related Content
-
A Novel CARM1-HuR Axis Involved in Muscle Differentiation and Plasticity Misregulated in Spinal Muscular AtrophySpinal muscular atrophy (SMA) is charact...
-
Targeted NGS Seen as Best When Screening for SMA and Its CarriersTargeted next generation sequencing (NGS...
-
Nonrespiratory Complications of Nusinersen-Treated Spinal Muscular Atrophy Type 1 PatientsBackground: Emergence of new treatments ...
-
Claire Altman Heine FoundationThe Claire Altman Heine Foundation (CAHF...
-
Vitamin K Deficiency Caused Blood Clotting Problem in SMA TeenAn adolescent male with spinal muscular ...
-
SMA: Considerations for Newborn Screening and Managementhttps://www.youtube.com/watch?v=FkshzHlc...
-
New Gene Therapy Transforms SMA (Spinal Muscular Atrophy)https://www.youtube.com/watch?v=C5Dl3iMv...