Neuromuscular

A specialist in the care of patients with spinal muscular atrophy reviews the CHERISH trial of nusinersen and examines the drug’s safety and efficacy profiles.

A key opinion leader in neurology highlights the value of a multidisciplinary approach to care for patients with spinal muscular atrophy in order to maximize a patient’s function through physical and medical therapy.

Patients with neurological sequelae from COVID-19 were significantly more likely to die before discharge (22.2%) compared with non-neuro-COVID patients (14.4%).

Many applicants referenced an early interest and exposure to neurology, often describing their first courses in neurology sparking their desire to learn more about how the brain works.

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The chief medical officer of Scholar Rock discussed the upcoming phase 3 trial for apitegromab in SMA and whether a successful trial would warrant FDA submission.

An overview of the diagnosis of spinal muscular atrophy, including prevalence, age of onset, and the role of genetic testing.

James Wymer, MD, provides a case overview of a 38-year-old female diagnosed with spinal muscular atrophy.

An expert neurologist, Arun S. Varadhachary, MD, PhD considers the diagnostic process for SMA and emphasizes the importance of early diagnosis to optimize care.

Arun S. Varadhachary, MD, PhD provides an overview of the case of a 28-year-old male with spinal muscular atrophy, describes the 4 main phenotypes of SMA, and considers how average age of onset impacts patient prognosis.

Basis for the approval was the SENZA-PDN randomized controlled trial, which showed the system’s ability to substantially sustain pain relief and improve health-related quality of life.

The chief medical officer of Scholar Rock discussed the greatest areas of concern for patients with SMA and why most of these issues exist in the diagnostic stage.

John Brandsema, MD, and Julie Parsons, MD, review the evolution of combination therapy in clinical practice for the management of SMA.

The effect observed in on ALSFRS-R at week 48 and progression-free survival further support the premise of greater treatment effect when masitinib is initiated earlier in the disease.

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John Brandsema, MD, comments on the importance of patient compliance to therapy to manage spinal muscular atrophy.

Experts in pediatric neurology share insights on the role of small molecule treatment for SMA through the recent FDA approval of risdiplam and discuss factors to consider when tailoring therapy for individual patients.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending July 15, 2021.

Since vaccine administration, the CDC has announced 100 preliminary cases of Guillain-Barré syndrome.

The chief medical officer of Scholar Rock described how the investigational apitegromab aims to fill some of the current unmet needs for patients with spinal muscular atrophy.

Efficacy results from a 109-week interim analysis showed improvement for patients treated with viltolarsen when compared to the matched DMD historical control group.

The chief medical officer of Scholar Rock detailed the additive benefits apitegromab has in treating SMA when used with previously approved nusinersen.

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John Brandsema, MD, leads the discussion on the concept of gene transfer for the management of SMA and the recent approval of onasemnogene abeparvovec-xioi.