Neuromuscular

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.

The director of the Center for Spinal Cord Injury Research and co-director of the Spinal Cord Injury Model System Center at Kessler Foundation discussed the secondary medical complications of spinal cord injury and how he and colleagues seek to alleviate them.

Study results show that deflazacort tops prednisone across multiple checkpoints in patients with Duchenne muscular dystrophy.

The drug is now approved to treat pediatric patients with both upper and lower limb spasticity, excluding spasticity caused by cerebral palsy.

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 as someone who experienced it.

New trials and research partnerships have launched with hopes to find answers for the terminal disease.