Standardized Tapering off Subcutaneous Immunoglobulin in Chronic Inflammatory Demyelinating Polyneuropathy.

Background: Attempting discontinuation of treatment in patients with chronic inflammatory demyelinating polyneuropathy (CIDP) is recommended. However, there is no evidence based regimen for tapering off subcutaneous immunoglobulin (SCIG). This trial investigated stepwise tapering off SCIG to detect remission and the lowest effective dosage.

Axial muscle involvement in patients with limb girdle muscular dystrophy type R9.

Introduction/aims: Limb girdle muscular dystrophy type R9 (LGMDR9) is characterized by progressive weakness of the shoulder and hip girdles. Involvement of proximal extremity muscles is well-described whereas information about axial muscle involvement is lacking. It is important to recognize the involvement of axial muscles to understand functional challenges for the patients.

Muscle involvement assessed by quantitative magnetic resonance imaging in patients with anoctamin 5 deficiency.

Objective: Using magnetic resonance imaging (MRI) and stationary dynamometry, the aim was to investigate the muscle affection in paraspinal muscles and lower extremities and compare the muscle affection in men and women with anoctamin 5 (ANO5) deficiency.

Methods: Seventeen patients (seven women) with pathogenic ANO5-mutations were included. Quantitative muscle fat fraction of back and leg muscles were assessed by Dixon MRI.

Patients With Becker Muscular Dystrophy Have Severe Paraspinal Muscle Involvement.

Paraspinal muscles are important for gross motor functions. Impairment of these muscles can lead to poor postural control and ambulation difficulty. Little knowledge exists about the involvement of paraspinal muscles in Becker muscular dystrophy.

Quantitative Muscle MRI as Outcome Measure in Patients With Becker Muscular Dystrophy-A 1-Year Follow-Up Study.

With the advent of emerging molecular therapies for muscular dystrophies, the need for knowledge about natural history course of such diseases is of utmost importance in the preparation for future trials. However, for Becker muscular dystrophy such knowledge is scarce. In this 1-year follow-up study, we examined disease progression in Becker muscular dystrophy by monitoring changes in MRI-assessed muscle fat fraction (FF) in axial and lower limb muscles and quantitative muscle strength of axial muscles.

Responsiveness of outcome measures in myotonic dystrophy type 1.

Objective: As myotonic dystrophy type 1(DM1) evolves slowly and interventional trials often have a short duration, responsive outcomes in DM1 are needed. The objective of this study was to determine the responsiveness of muscle strength, balance, and functional mobility measurements after a 1-year follow-up period in individuals with DM1.

Methods: Sixty-three adults with noncongenital DM1 completed the following assessments at baseline and at 1-year follow-up: Handheld dynamometry (lower limbs), stationary dynamometry (lower limbs), step test, timed-up-and-go test (TUG), modified clinical test of sensory integration and balance (mCTSIB), feet-together stance, tandem stance, one-leg stance, 10-meter walk test, and sit-to-stand test.

Intrarater reliability and validity of outcome measures in myotonic dystrophy type 1.

Objective: To investigate intrarater reliability and concurrent and construct validity of muscle strength, balance, and functional mobility measures in individuals with noncongenital myotonic dystrophy type 1 (DM1).

Methods: Seventy-eight adults with noncongenital DM1 participated in visit 1, and 73 of the them participated in visit 2 separated by 1 to 2 weeks. The assessments consisted of muscle strength tests with handheld dynamometry (HHD) and stationary dynamometry in the lower limb.

Physical activity in myotonic dystrophy type 1.

Background: Physical inactivity is associated with lifestyle diseases and exercise of moderate intensity seems beneficial in DM1, but knowledge about physical activity and predictors of physical activity in individuals with myotonic dystrophy type 1 (DM1) is limited. The objective of this study is to assess physical activity and predictors of physical activity in individuals with DM1.

Methods: Sixty-seven adults with DM1 and 39 healthy adults were recruited.

Exercise therapy for muscle and lower motor neuron diseases.

Muscle and lower motor neuron diseases share a common denominator of perturbed muscle function, most often related to wasting and weakness of muscles. This leads to a number of challenges, such as restricted mobility and respiratory difficulties. Currently there is no cure for these diseases.