Neuromuscular Disorders

Latest News

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.

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.

Virtual Reality Technologies Improve Outcomes in Duchenne Muscular Dystrophy

A team of researchers sought to evaluate the potential of virtual reality technologies for rehabilitation of patients with Duchenne muscular dystrophy.

Antisense Oligonucleotide Reduces Pathologic Regeneration in Duchenne Muscular Dystrophy

Antisense oligonucleotide golodirsen may be associated with reduced pathologic regeneration in boys with Duchenne muscular dystrophy.