February 15, 2011Archives of NeurologyOpen Access

Observational Study of Spinal Muscular Atrophy Type 2 and 3

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Population

65 participants with spinal muscular atrophy (SMA) types 2 and 3, aged 20 months to 45 years

Design

Cohort

Follow-up

12 months

Authors

PKPetra KaufmannUniversity of the Sciences

Discussion

Member takes

Implication

Supports stable course in SMA types 2/3 over 12 months; hypothesis-generating for trials targeting improvement rather than stabilization.

Structured PICO

Population

65 participants with spinal muscular atrophy (SMA) types 2 and 3, aged 20 months to 45 years

Intervention

Observational study (collection of demographic and medical history information, determination of SMN2 copy number)

Outcome

Change over 12 months in clinical outcomes including measures of motor function (Gross Motor Function Measure and expanded Hammersmith Functional Motor Scale), pulmonary function (forced vital capacity), and muscle strength (myometry)surrogate

SMA types 2 and 3 have a relatively stable clinical course over 12 months, indicating that future clinical trials should be designed to measure improvement rather than stabilization of disease progression.

Cite This Study

Petra Kaufmann (2011) studied this question.

synapsesocial.com/papers/6a8666e46fcdf5c7461b2ec5 https://doi.org/10.1001/archneurol.2010.373

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Observational Study of Spinal Muscular Atrophy Type 2 and 3

Discussion

Member takes

Implication

Structured PICO

Cite This Study

Observational Study of Spinal Muscular Atrophy Type 2 and 3

Discussion

Member takes

Implication

Structured PICO

Cite This Study

Also Consider

Also Consider