Neuromuscular

Advocacy organization Cure SMA provides guidance for health care providers caring for patients with SMA during the ongoing COVID-19 pandemic.

The drug is indicated in patients with a confirmed mutation in the DMD gene that is amenable to exon 53 skipping.

The co-director of the Neurological Clinical Research Institute at Massachusetts General Hospital spoke to findings of the phase 2 CENTAUR trial of AMX0035 in amyotrophic lateral sclerosis.

After receiving fast track designation by the FDA in January 2018, pegunigalsidase alfa now has a PDUFA date scheduled for January 27, 2021.

Adding to a flurry of pipeline movement since July, 2 more rare disease agents—VTA-110 for DMD and arimoclomol for NPC—have made positive steps in their clinical development recently.

The agent, marketed under the brand name Evrysdi, is the first at-home therapy indicated for treatment of adults and children with SMA.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending August 7, 2020.

The co-director of the Neurological Clinical Research Institute at Massachusetts General Hospital spoke to the need for more collaborative efforts in ALS research.

An overview of the tweet chat on mental health in neurological disorders, featuring highlighted contributions from the medical community.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending July 31, 2020.

The co-director of the Neurological Clinical Research Institute at Massachusetts General Hospital discussed how the investigational agent might fit into the landscape of amyotrophic lateral sclerosis treatment as it progresses through the pipeline.

Join NeurologyLive and the Women Neurologist Group on Twitter to discuss the importance of mental health and wellness in neurologic disorders.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending July 24, 2020.

The RESPOND study steering committee member and pediatric neuromuscular neurologist at Children’s Hospital of The King’s Daughters discussed the goals of the phase 4 trial of nusinersen in SMA.

The vice president and head of the Neuromuscular Development Unit at Biogen, and study coauthor, discussed the implications of recently published data on tofersen and the ongoing work in phase 3.

The vice president and head of the Neuromuscular Development Unit at Biogen, and study coauthor, offered insight into the findings of a phase 1/2 trial of the SOD1-targeted antisense oligonucleotide.

The Clinigen Group’s ALS treatment is currently being investigated in a phase 2 trial that is expected to conclude in September 2021.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending July 17, 2020.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending July 10, 2020.

Cure SMA gives an overview of the recent 2020 Virtual SMA Conference, with highlights on new data and poster sessions.

The latest program from Cure SMA aims to better understand the capacity of spinal muscular atrophy centers in the US and to continue to carry out effective clinical trials.

In the phase 1/2 study, the Biogen agent showed a favorable reduction of SOD1 concentrations compared to placebo, particularly with the 100-mg dose, and is being assessed in an ongoing phase 3 study, VALOR.

The National Institutes of Health program, dubbed Accelerating Leading-edge Science in ALS, or ALS2, is set to invest $25M over 5 years for exceptionally creative and riskier research.

One of the first case-series studies that analyzed children with COVID-19 suggest that SARS-CoV-2 should be considered for differential diagnosis in children presenting with new neurologic symptoms and splenium signal changes on MRI.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending July 3, 2020.