Neuromuscular

Darryl De Vivo, MD, emphasized that while there's been a tremendous advance in the field of SMA, it’s important to continue tinkering around the margins to make the effective treatment increasingly better.

The Sidney Carter Professor of Neurology at Columbia University Medical Center spoke about the 127 year history of spinal muscular atrophy and how basic science has led to exciting developments.

Since the inception of the idea more than 3 decades ago and its initial development 20 years later, Sarepta Therapeutics’ micro-dystrophin gene therapy has now made its way to human trials.

The gene therapy is also being assessed for long-term safety data, as well as an additional delivery method.

A novel mesenchymal stem cell therapy produced intriguing phase II data and has moved into a randomized, pivotal phase III clinical trial.

Patisiran maintained improvements in mNIS+7 and rapidly halted neurologic disease progression in hATTR amyloidosis.

Subcutaneous immunoglobulin is an effective and well-tolerated alternative to intravenous immunoglobulin for myasthenia gravis.

Eculizumab showed sustained activity and tolerability through 3 years for generalized myasthenia gravis.

In a small trial, the AAVrh74.MHCK7.micro-dystrophin therapy has shown high levels of transduced micro-dystrophin expression.

The anti-inflammatory agent is also being explored in progressive MS, with phase II results reading out earlier this year.

The relationship between disease stage and behavior is important given the strength of the relationship relative to cognition and its negative impact on patients and caregivers.

An emerging class of cancer drugs may help treat brain disorders such as amyotrophic lateral sclerosis frontotemporal dementia.

Also known as S48168, it was previously granted Orphan Drug designation as well as a Rare Pediatric Disease designation in 2015 for Duchenne Muscular Dystrophy.

The approval was based on bioavailability studies which compared the tablet formulation of riluzole to the oral suspension formulation.

The director of the electromyography laboratory and a professor of neurology at Cedars-Sinai discussed the misdiagnosis of the rare condition.

The director of the electromyography laboratory and a professor of neurology at Cedars-Sinai spoke about the treatment options for CIDP.

Jeffrey Allen, MD, spoke about the breakthrough barrier in CIDP and the importance of the guidelines for diagnosis.

If your patient is a construction worker, bus driver, gas station attendant, or mechanic, take notice: “This type of exposure deserves more attention and study as we work to develop a better understanding of what causes ALS,” notes the author of a recent study.

Results provide Class IV evidence that nusinersen is safe and efficient on motor symptoms in patients older than 7 months of age.

What are the best available options for patients with CIDP, and what is still missing?

A subcutaneous intrathecal catheter delivery system has proven safe and tolerable in a preliminary investigation in patients with SMA.

The director of the ALS Clinic at Massachusetts General Hospital pointed to the robust pipeline and meaningful gains in knowledge about the disease as reason to be hopeful.

The mesenchymal stem cell therapy passed an interim safety analysis for the first 31 patients with amyotrophic lateral sclerosis.

The designation is backed by positive interim data from ASPIRO, which has demonstrated significant improvements in neuromuscular and respiratory function at week 24.

Several clinical trials are examining the role of stem cells, inflammation, and other pathways in ALS.