The Effects of ICD Coding Transition on Neurologic Diagnoses
The transition from the International Classification of Disease (ICD)-9 to ICD-10 coding system may impact estimates of neurologic disease prevalence.
Neuromuscular Disorders
Myrbetriq Approved for Pediatric Neurogenic Detrusor Overactivity
Both formulations are indicated for patients aged 3 years and older; however, the tablet formulation is specifically approved for patients weighing 35kg or more.
Risdiplam Shows Promise for Infants With Type 1 Spinal Muscular Atrophy
Investigators sought to examine the safety, pharmacokinetics, and pharmacodynamics of risdiplam in infants with type 1 spinal muscular atrophy.
Zolgensma Benefits Observed in Presymptomatic SMA Patients
The phase 3 SPR1NT trial is an ongoing open-label, single-arm study evaluating onasemnogene in presymptomatic patients (≤6 weeks of age) with a genetic diagnosis of SMA and 2 or 3 copies of survival motor neuron 2 gene.
Long-Term Data Demonstrate Sustained Efficacy of Risdiplam in Spinal Muscular Atrophy
In Part 2 of the trial, patients 2-25 years of age received either risdiplam for 24 months or placebo for 12 months followed by risdiplam for 12 months.
FDA to Review Efgartigimod for Generalized Myasthenia Gravis
Efgartigimod, an Fc receptor antagonist, reduces disease-causing immunoglobulin G (IgG) antibodies and blocks the IgG recycling process.
Improved Muscle Quality in Duchenne Muscular Dystrophy With SRP-9001
Researchers sought to discover whether SRP-9001 would aid large muscle groups, as measured with quantitative MRI and spectroscopy.
Amondys 45 Approved for Duchenne Muscular Dystrophy
Treatment with casimersen led to a statistically significant increase in dystrophin production in skeletal muscle from baseline to week 48.
New Treatment Recommendations for Myasthenia Gravis
A panel of international experts provided new evidence-based recommendations for the management of myasthenia gravis, including new guidance for thymectomy and medical treatment.
Caring for Patients With Duchenne Muscular Dystrophy During COVID-19 Pandemic
Tori Rodriguez, MA, LPC, discusses the progression of care for patients with Duchenne muscular dystrophy during the COVID-19 pandemic.
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