Neuromuscular

Efforts to cut through regulatory red tape will help ensure earlier access to potentially lifesaving treatments.

Neurology News Network for the week ending August 22, 2020.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending August 21, 2020.

Qutenza becomes the first topical treatment with a TRPV1 agonist indicated for the treatment of neuropathic pain associated with diabetic peripheral neuropathy.

The co-director of the Neurological Clinical Research Institute at Massachusetts General Hospital offered her perspective on the expected developments for ALS care in next few years, and where AMX0035 might fit in that landscape.

The presence of laryngeal movement disorders—identified in 93% of the MSA group—may serve as a diagnostic biomarker of MSA independent of phenotype, disease duration, or patient age.

Neurology News Network for the week ending August 15, 2020.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending August 14, 2020.

"Mind Moments," a podcast from NeurologyLive, brings you an exclusive interview with Richard Finkel, MD.

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.