Key Terminologies in Neuromuscular Research
1. Propensity Score Matching (PSM)
A statistical method used to balance groups in observational studies, reducing bias by matching individuals with similar characteristics. This technique improves the validity of comparisons, especially in studies where randomization isn't feasible.
2. North Star Ambulatory Assessment (NSAA) & North Star Assessment
These are clinical tools used to measure motor abilities in patients with neuromuscular disorders. The NSAA, for example, is frequently used in Duchenne Muscular Dystrophy (DMD) to assess functions like standing, walking, and stair climbing over time.
3. Sarcoglycanopathy
A type of limb-girdle muscular dystrophy caused by mutations in the sarcoglycan proteins. These proteins are crucial for maintaining muscle integrity, and their dysfunction leads to progressive muscle weakness, especially in the shoulders and hips.
4. Cross-sectional Study
A research design that examines data collected from a population at one point in time. It provides insights into a specific condition or characteristic without following subjects over time.
5. Bulbar Palsy
A condition characterized by weakness in the muscles controlled by the brainstem, impacting functions like speaking and swallowing. It is commonly seen in diseases such as ALS and certain muscular dystrophies.
Figure 1 : DOI: 10.1055/s-0043-1772833
Figure 2 : World Muscle Society, October 2020, Virtual Poster # 286