Neuromuscular

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.

Part 2 of the pivotal FIREFISH clinical trial met its primary end point in infants aged 1 to 7 months with Type 1 spinal muscular atrophy, with statistically significant and medically meaningful motor milestone improvements.

Use of rimabotulinumtoxinB (Myobloc; US WorldMeds) result in statistically significant reductions in sialorrhea compared to placebo with both 2500 U and 3500 U doses.

The newly created 28-question assessment showed improved item targeting compared with the current standard Amyotrophic Lateral Sclerosis Functional Rating Scale.

In this Peer Exchange, experts discussed current SMA therapies, what they have learned, and what the future holds for treatment of this devastating disease.

The approvals introduced several new classes of drugs, including the ditans and gepants in migraine, as well as novel formulations that can ease the administration of treatments for Parkinson disease and epilepsy.

The director of the Jefferson Headache Center at Thomas Jefferson University detailed the most notable achievements from 2019, and what to expect in the coming year.

Catch up on some of our most popular Peer Exchange panels with experts in the field, including discussions on epilepsy, migraine, multiple sclerosis, and more.

After promising results in prior phase 2 study, Stealth BioTherapeutics’ elampretide has failed to show change in the 6-minute walk test and Primary Mitochondrial Myopathy Symptom Assessment total fatigue score in the MMPOWER-3 trial.

The behavioral director at the Angelman Syndrome Clinic at Massachusetts General Hospital offered insight into the challenges in studying a drug, such as gaboxadol, in Angelman syndrome.

An in-depth analysis of the genetics of DMD and disease modifiers such as the NF-kB pathway in patients with Duchenne muscular dystrophy.

The behavioral director at the Angelman Syndrome Clinic at Massachusetts General Hospital discussed the phase 3 NEPTUNE trial of gaboxadol, also known as OV101.

Neurology News Network for the week ending December 21, 2019.