Neuromuscular

In addition to significant improvements compared to placebo on Quantitative QMG scores, zilucoplan improved patients' activities of daily living and quality of life scores.

The Prometra II offers a pressure-driven, valve-gated delivery mechanism that allows for novel programming modes of intermittent flow followed by periods of no flow—a feature unique to this device.

The announcement formally connects Bcureful and the Tuberous Sclerosis Alliance together after working closely together for over the past 8 years.

The revision accounts for data from the NURTURE trial showing that patients with 3 copies of SMN2 greatly benefit from early treatment.

The Tuberous Sclerosis Alliance has announced updates to access of everolimus, a drug used to treat several conditions associated with tuberous sclerosis complex.

Neurofilament light chain has emerged as a biomarker with utility across the breadth of neurology, but just how much can it actually help?

The Nippon Shinyaku investigational DMD agent has a Prescription Drug User Fee Act date within the third quarter of 2020.

New findings suggest a strong association between certain lower extremity biomarkers and measures of clinical function in patients with Duchenne muscular dystrophy.

The biotech company plans to roll out 2 clinical trials for patients with amyotrophic lateral sclerosis following the orphan drug designation for PrimeC.

Neurology News Network for the week ending February 8, 2020.

The study is the first placebo-controlled trial to include non-ambulant adults with spinal muscular atrophy type 2 or 3.

Interim analysis compared ataluren in a real-world setting from the STRIDE registry with a matched cohort; the final data from the STRIDE registry are expected in 2025.

Join NeurologyLive and the Women Neurologists Group on Twitter to celebrate women in neurology on National Women Physicians Day.

New study data showed nerve ultrasound had a sensitivity of 97.4% and specificity of 78.9%, with an added value in the detection of treatment-responsive chronic inflammatory neuropathy of 21.1%.

Subgroups and treatment-related changes can be identified with comparative cerebrospinal fluid proteomic analysis in adult patients with spinal muscular atrophy who are treated with nusinersen.

The first trial of its kind in amyotrophic lateral sclerosis will first examine 3 drugs and add additional treatments as more become available.