Neuromuscular

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.

CENTAUR trial principal investigator Sabrina Paganoni, MD, PhD, discussed details of the phase 2 trial that helped investigators have the greatest chance of seeing a measurable treatment effect in patients with ALS.

Top-line results from the phase 2 CENTAUR trial showed a statistically significant slowing of ALS disease progression in patients treated with AMX0035.

The CEO and Chairman of the Board of Directors at INSIGHTEC offered his perspective on how technologies such as focused ultrasound can help shape the future of neurologic care.

Wave Life Sciences has discontinued its suvodirsen clinical trials after investigators saw no change from baseline in either of the dose groups.

The CEO of INSIGHTEC offered insight into what challenges in the neurologic space the company plans to tackle in the coming years, and why 2020 marks the start of the “Century of the Brain.”

A survey by the ALS Association found that 26% of patients reported having a PEG tube, which are typically inserted to allow for continued administration of nutrition, fluids, and medications as the disease progresses.

After receiving expedited review after receiving a Complete Response Letter in August, the therapy was approved based on data showing a statistically significant increase in dystrophin production in skeletal muscle.

Laboratories in the US are now authorized to add the GSP Neonatal Creatine Kinase-MM kit to their newborn screening panels if they choose to do so.

Although there are gaps in knowledge, investigators see potential in telemedicine for other neurologic disorders beyond stroke.

Neurology News Network for the week ending November 30, 2019.

The novel formulation of edaravone, which was approved in May 2017, will be assessed in a cohort of 150 patients with amyotrophic lateral sclerosis for 48 weeks of treatment.

The use of cerebrospinal neurofilament light chain and TAR DNA‐binding protein 43 in tandem may offer stronger biomarkers for amyotrophic lateral sclerosis diagnosis, with NfL also offering a measure of disease progression.