Neuromuscular

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.

The FDA set a PDUFA date for risdiplam of May 24, 2020. The full 12-month data from the SUNFISH trial are expected to be presented at an upcoming medical meeting.

The approval is supported by several studies which demonstrated the drug’s bioequivalence to Rilutek, the liquid riluzole formulation marketed by Sanofi.

The mesenchymal stem cell-neurotrophic factor cells met the primary safety end point and displayed early but promising signs of efficacy, prompting excitement among investigators for moving forward with phase 3 study.

The spine surgeon in the Center for Spine Health at Cleveland Clinic discussed the importance of patient-reported outcomes for patients who are undergoing spine surgery.

The spine surgeon in the Center for Spine Health at Cleveland Clinic spoke about the improvements in minimally invasive surgery and the areas ripe for innovation.

Neurology News Network for the week ending November 16, 2019.

This is the third safety incident in the IGNITE DMD clinical trial that has resulted in a clinical hold since its inception in 2017.

Patients with spinal muscular atrophy types 2 or 3 saw improvements in motor function after receiving risdiplam; data will be presented at an upcoming medical congress.

The robotic platform used in various neurosurgeries was identified for a class 1 recall due to a software error which can lead to robotic arm malfunction.

The phase 2/3 trials will end after a futility analysis suggested that the drug is not likely to show a clinical benefit.

The director of the Center for Spinal Cord Injury Research and co-director of the Spinal Cord Injury Model System Center at Kessler Foundation shared his unique perspective on spinal cord injury and the work he and colleagues are doing to alleviate its challenges.

Neurology News Network for the week ending October 28, 2019.

The chief medical executive at The University of Texas MD Anderson Cancer Center is expected to be named the next commissioner of the FDA by President Donald Trump.

The drug, which is currently approved for single-dose intravenous administration in patients with SMA type 1, is currently being investigated as intrathecal therapy in SMA type 2.