FDA to Review DMD Treatment Golodirsen
Duchenne affects an estimated 1 in approximately every 3,500-5,000 males born worldwide.
Neuromuscular Disorders
Thymectomy + PDN Beneficial for Non-Thymomatous Myasthenia Gravis
For patients with non-thymomatous myasthenia gravis, thymectomy plus prednisone confers benefits versus prednisone alone at 5 years.
Mexiletine Associated With Better Reduction in Muscle Stiffness Than Placebo in Nondystrophic Myotonia
Findings support the efficacy of mexiletine and the potential value of N-of-1 trials for assessing interventions in some chronic rare diseases.
Severe Fatigue Predicts Worsening Disability in Patients With MS
Researchers investigated a link between fatigue and disease worsening in patients with multiple sclerosis.
Movement Disorders Highly Prevalent in Early Multiple Sclerosis
Investigators observed a wide variety of movement disorders — including restless legs syndrome, tremor, and tonic spasms — associated with early demyelinating diseases.
Risk for Conversion of MS Varies With Different Therapies
For relapsing-remitting multiple sclerosis (MS), the risk for conversion to secondary progressive MS is lower with initial treatment with fingolimod, alemtuzumab, or natalizumab versus glatiramer acetate or interferon beta.
Resting-State MEG May Indicate MS-Related Cognitive Decline
Investigators observed a clinically relevant slowing of neuronal activity in patients with MS in the parietotemporal cortical areas and the thalamus significantly associated with cognitive impairment.
Odor Discrimination and Identification Linked to Neurodegeneration in MS
Researchers evaluated patients with MS to determine whether their unique problems with their sense of smell could be due to inflammatory activity or neurodegeneration.
Addressing Cognitive Impairment in Pediatric MS: Expert Q&A
Cognitive reserve may attenuate the impact of MS on cognitive function in some patients.
Ocrelizumab Reduces Upper Extremity Disability Progression Risk in PPMS
Investigators observed that monoclonal antibody ocrelizumab may reduce the risk for upper extremity disability progression in patients with primary progressive multiple sclerosis.
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